Donald Barry Kohn

Title(s)Professor, MIMG
SchoolMedicine
vCardDownload vCard
    Other Positions
    Title(s)Professor, Molecular and Medical Pharmacology

    Title(s)Professor, Pediatrics


    Collapse Research 
    Collapse Research Activities and Funding
    Disseminating Curative Biological Therapies for Rare Pediatric Diseases
    NIH U01TR001814Sep 16, 2016 - Jun 30, 2021
    Role: Co-Principal Investigator
    A Phase II/III Clinical Trial of Gene Therapy for ADA-Deficient SCID
    NIH/NIAID U01AI122260Mar 1, 2016 - Feb 28, 2022
    Role: Principal Investigator
    EFS-ADA Lentiviral Vector Transduction of Bone Marrow CD34+ Cells for ADA-SCID
    NIH U01AI100801Aug 1, 2012 - Jul 31, 2018
    Role: Principal Investigator
    Primary Immune Deficiency Treatment Consortium
    NIH U54AI082973Sep 12, 2009 - Aug 31, 2024
    Role: Co-Principal Investigator
    In Vivo ADA Gene Delivery for the Treatment of SCID
    NIH R01AI074043Jun 1, 2007 - May 31, 2013
    Role: Principal Investigator
    Training in Developmental Hematology
    NIH T32HL086345Apr 1, 2007 - May 31, 2023
    Role: Principal Investigator
    Clinical Trial Planning Grant: Lentiviral Vector for HIV-1
    NIH R34AI069947May 15, 2006 - Oct 30, 2007
    Role: Principal Investigator
    MND-ADA Transduced CD34 Cells for ADA-SCID
    NIH R01FD003005Sep 30, 2005 - Feb 28, 2013
    Role: Principal Investigator
    Conference--American Society of Gene Therapy
    NIH R13HL078311Jul 1, 2004 - Jun 30, 2009
    Role: Principal Investigator
    Gene Therapy Using Hematopoietic Stem Cells
    NIH P01HL073104Apr 1, 2003 - Jun 30, 2015
    Role: Principal Investigator
    GENE EXPRESSION IN BETA-CELLS BY LENTIVIRAL VECTORS
    NIH R21DK062649Jul 1, 2002 - Jun 30, 2004
    Role: Principal Investigator
    LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    NIH R01AI052798Jun 1, 2002 - May 31, 2007
    Role: Principal Investigator
    MODIFIED RETROVIRAL VECTORS FOR HSC GENE THERAPY
    NIH R01DK056287Aug 1, 1999 - Jul 31, 2003
    Role: Principal Investigator
    GENE THERAPY FOR HURLERS DISEASE
    NIH R01DK054566Sep 5, 1998 - May 31, 2002
    Role: Principal Investigator
    IN VIVO MODEL OF HUMAN STEM CELL GENE THERAPY
    NIH R01DK048700Sep 30, 1994 - Aug 31, 1998
    Role: Principal Investigator
    MODIFIED RETROVIRAL VECTORS FOR STABLE GENE EXPRESSION
    NIH R01DK049000Sep 30, 1994 - Aug 31, 1997
    Role: Principal Investigator
    GLUCOCEREBROSIDASE GENE EXPRESSION--HEMATOPOIETIC CELLS
    NIH R01DK042694Sep 1, 1991 - Aug 31, 1996
    Role: Principal Investigator
    RETROVIRAL VECTOR-MEDIATED TRANSFER OF NEF GENE
    NIH R01AI029125Sep 30, 1989 - Jul 31, 1993
    Role: Principal Investigator

    Collapse ORNG Applications 
    Collapse Clinical Trials

    Collapse Bibliographic 
    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
    Newest   |   Oldest   |   Most Cited   |   Most Discussed   |   Timeline   |   Field Summary   |   Plain Text
    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. Clin Immunol. 2024 Feb 15; 261:109942. Abraham RS, Basu A, Heimall JR, Dunn E, Yip A, Kapadia M, Kapoor N, Satter LF, Buckley R, O'Reilly R, Cuvelier GDE, Chandra S, Bednarski J, Chaudhury S, Moore TB, Haines H, Dávila Saldaña BJ, Chellapandian D, Rayes A, Chen K, Caywood E, Chandrakasan S, Lugt MTV, Ebens C, Teira P, Shereck E, Miller H, Aquino V, Eissa H, Yu LC, Gillio A, Madden L, Knutsen A, Shah AJ, DeSantes K, Barnum J, Broglie L, Joshi AY, Kleiner G, Dara J, Prockop S, Martinez C, Mousallem T, Oved J, Burroughs L, Marsh R, Torgerson TR, Leiding JW, Pai SY, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Cowan MJ, Puck J, Dvorak CC, Haddad E. PMID: 38367737.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCellsPHPublic Health
    2. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study. J Allergy Clin Immunol. 2024 Jan 28. Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Deal CL, Kapoor N, Murguía-Favela L, Falcone EL, Prasad VK, Touzot F, Bleesing JJ, Chandrakasan S, Heimall JR, Bednarski JJ, Broglie LA, Chong HJ, Kapadia M, Prockop S, Dávila Saldaña BJ, Schaefer E, Bauchat AL, Teira P, Chandra S, Parta M, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Leiding JW. PMID: 38290608.
      View in: PubMed   Mentions:    Fields:    
    3. What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity. J Allergy Clin Immunol Pract. 2024 Jan 19. Mudde ACA, Kuo CY, Kohn DB, Booth C. PMID: 38246560.
      View in: PubMed   Mentions:    Fields:    
    4. Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD. Blood. 2023 12 14; 142(24):2105-2118. Leiding JW, Arnold DE, Parikh S, Logan B, Marsh RA, Griffith LM, Wu R, Kidd S, Mallhi K, Chellapandian D, Si Lim SJ, Grunebaum E, Falcone EL, Murguia-Favela L, Grossman D, Prasad VK, Heimall JR, Touzot F, Burroughs LM, Bleesing J, Kapoor N, Dara J, Williams O, Kapadia M, Oshrine BR, Bednarski JJ, Rayes A, Chong H, Cuvelier GDE, Forbes Satter LR, Martinez C, Vander Lugt MT, Yu LC, Chandrakasan S, Joshi A, Prockop SE, Dávila Saldaña BJ, Aquino V, Broglie LA, Ebens CL, Madden LM, DeSantes K, Milner J, Rangarajan HG, Shah AJ, Gillio AP, Knutsen AP, Miller HK, Moore TB, Graham P, Bauchat A, Bunin NJ, Teira P, Petrovic A, Chandra S, Abdel-Azim H, Dorsey MJ, Birbrayer O, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM. PMID: 37562003; PMCID: PMC10862239.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    5. Gene therapy for adenosine deaminase severe combined immune deficiency-An unexpected journey of four decades. Immunol Rev. 2024 Mar; 322(1):148-156. Kohn DB. PMID: 38033164.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    6. Successes and challenges in clinical gene therapy. Gene Ther. 2023 Nov; 30(10-11):738-746. Kohn DB, Chen YY, Spencer MJ. PMID: 37935854; PMCID: PMC10678346.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    7. Busulfan and subsequent malignancy: An evidence-based risk assessment. Pediatr Blood Cancer. 2024 Jan; 71(1):e30738. Long-Boyle JR, Kohn DB, Shah AJ, Spencer SM, Sevilla J, Booth C, López Lorenzo JL, Nicoletti E, Shah A, Reatz M, Matos J, Schwartz JD. PMID: 37856098.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    8. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. J Allergy Clin Immunol. 2024 01; 153(1):287-296. Eissa H, Thakar MS, Shah AJ, Logan BR, Griffith LM, Dong H, Parrott RE, O'Reilly RJ, Dara J, Kapoor N, Forbes Satter L, Chandra S, Kapadia M, Chandrakasan S, Knutsen A, Jyonouchi SC, Molinari L, Rayes A, Ebens CL, Teira P, Dávila Saldaña BJ, Burroughs LM, Chaudhury S, Chellapandian D, Gillio AP, Goldman F, Malech HL, DeSantes K, Cuvelier GDE, Rozmus J, Quinones R, Yu LC, Broglie L, Aquino V, Shereck E, Moore TB, Vander Lugt MT, Mousallem TI, Oved JH, Dorsey M, Abdel-Azim H, Martinez C, Bleesing JH, Prockop S, Kohn DB, Bednarski JJ, Leiding J, Marsh RA, Torgerson T, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Heimall J. PMID: 37793572.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    9. Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease. J Allergy Clin Immunol. 2023 12; 152(6):1619-1633.e11. Chandrasekaran P, Han Y, Zerbe CS, Heller T, DeRavin SS, Kreuzberg SA, Marciano BE, Siu Y, Jones DR, Abraham RS, Stephens MC, Tsou AM, Snapper S, Conlan S, Subramanian P, Quinones M, Grou C, Calderon V, Deming C, Leiding JW, Arnold DE, Logan BR, Griffith LM, Petrovic A, Mousallem TI, Kapoor N, Heimall JR, Barnum JL, Kapadia M, Wright N, Rayes A, Chandra S, Broglie LA, Chellapandian D, Deal CL, Grunebaum E, Lim SS, Mallhi K, Marsh RA, Murguia-Favela L, Parikh S, Touzot F, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Kang EM, Malech HL, Segre JA, Bryant CE, Holland SM, Falcone EL. PMID: 37659505.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    10. Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells. ACS Appl Mater Interfaces. 2023 Sep 06; 15(35):41299-41309. Frost IM, Mendoza AM, Chiou TT, Kim P, Aizenberg J, Kohn DB, De Oliveira SN, Weiss PS, Jonas SJ. PMID: 37616579; PMCID: PMC10485797.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    11. Biodistribution of lentiviral transduced adipose-derived stem cells for "ex-vivo" regional gene therapy for bone repair. Gene Ther. 2023 Dec; 30(12):826-834. Bell JA, Collon K, Mayfield C, Gallo MC, Chang SW, Sugiyama O, Tang AH, Hollis R, Chopra S, Kohn DB, Lieberman JR. PMID: 37568039.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimalsCells
    12. Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium. Lancet. 2023 07 08; 402(10396):129-140. Thakar MS, Logan BR, Puck JM, Dunn EA, Buckley RH, Cowan MJ, O'Reilly RJ, Kapoor N, Satter LF, Pai SY, Heimall J, Chandra S, Ebens CL, Chellapandian D, Williams O, Burroughs LM, Saldana BD, Rayes A, Madden LM, Chandrakasan S, Bednarski JJ, DeSantes KB, Cuvelier GDE, Teira P, Gillio AP, Eissa H, Knutsen AP, Goldman FD, Aquino VM, Shereck EB, Moore TB, Caywood EH, Lugt MTV, Rozmus J, Broglie L, Yu LC, Shah AJ, Andolina JR, Liu X, Parrott RE, Dara J, Prockop S, Martinez CA, Kapadia M, Jyonouchi SC, Sullivan KE, Bleesing JJ, Chaudhury S, Petrovic A, Keller MD, Quigg TC, Parikh S, Shenoy S, Seroogy C, Rubin T, Decaluwe H, Routes JM, Torgerson TR, Leiding JW, Pulsipher MA, Kohn DB, Griffith LM, Haddad E, Dvorak CC, Notarangelo LD. PMID: 37352885; PMCID: PMC10386791.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansPHPublic Health
    13. Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. Cell. 2023 03 30; 186(7):1398-1416.e23. McAuley GE, Yiu G, Chang PC, Newby GA, Campo-Fernandez B, Fitz-Gibbon ST, Wu X, Kang SL, Garibay A, Butler J, Christian V, Wong RL, Everette KA, Azzun A, Gelfer H, Seet CS, Narendran A, Murguia-Favela L, Romero Z, Wright N, Liu DR, Crooks GM, Kohn DB. PMID: 36944331.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansAnimalsCells
    14. Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials. Viruses. 2023 03 09; 15(3). Segura EER, Ayoub PG, Hart KL, Kohn DB. PMID: 36992422; PMCID: PMC10054523.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    15. β-Thalassemia: all about that base, no cutting. Blood. 2023 03 09; 141(10):1098-1099. Ayoub PG, Kohn DB. PMID: 36893006.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    16. Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression. Blood. 2023 03 02; 141(9):1007-1022. Wong RL, Sackey S, Brown D, Senadheera S, Masiuk K, Quintos JP, Colindres N, Riggan L, Morgan RA, Malech HL, Hollis RP, Kohn DB. PMID: 36332160; PMCID: PMC10163279.
      View in: PubMed   Mentions: 4     Fields:    Translation:Animals
    17. Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Hum Gene Ther. 2023 02; 34(3-4):83-89. McDonald CL, Qasba P, Anderson DG, Bao G, Colvin RA, Kohn DB, Malik P, Mitchell MJ, Pu WT, Rawlings DJ, Williams DA, Flotte TR. PMID: 36794978; PMCID: PMC10081695.
      View in: PubMed   Mentions:    Fields:    
    18. Updated Management Guidelines for Adenosine Deaminase Deficiency. J Allergy Clin Immunol Pract. 2023 06; 11(6):1665-1675. Grunebaum E, Booth C, Cuvelier GDE, Loves R, Aiuti A, Kohn DB. PMID: 36736952.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    19. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. J Clin Oncol. 2023 04 20; 41(12):2227-2237. Eapen M, Brazauskas R, Williams DA, Walters MC, St Martin A, Jacobs BL, Antin JH, Bona K, Chaudhury S, Coleman-Cowger VH, DiFronzo NL, Esrick EB, Field JJ, Fitzhugh CD, Kanter J, Kapoor N, Kohn DB, Krishnamurti L, London WB, Pulsipher MA, Talib S, Thompson AA, Waller EK, Wun T, Horowitz MM. PMID: 36623245; PMCID: PMC10448940.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    20. Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials. Mol Ther. 2023 03 01; 31(3):801-809. Cornetta K, Yao J, House K, Duffy L, Adusumilli PS, Beyer R, Booth C, Brenner M, Curran K, Grilley B, Heslop H, Hinrichs CS, Kaplan RN, Kiem HP, Kochenderfer J, Kohn DB, Mailankody S, Norberg SM, O'Cearbhaill RE, Pappas J, Park J, Ramos C, Ribas A, Rivière I, Rosenberg SA, Sauter C, Shah NN, Slovin SF, Thrasher A, Williams DA, Lin TY. PMID: 36518078; PMCID: PMC10014217.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCells
    21. Meeting FDA Guidance recommendations for replication-competent virus and insertional oncogenesis testing. Mol Ther Methods Clin Dev. 2023 Mar 09; 28:28-39. Cornetta K, Lin TY, Pellin D, Kohn DB. PMID: 36588821; PMCID: PMC9791246.
      View in: PubMed   Mentions: 5  
    22. Improving Lentiviral Transduction of Human Adipose-Derived Mesenchymal Stem Cells. Hum Gene Ther. 2022 12; 33(23-24):1260-1268. Collon K, Gallo MC, Bell JA, Chang SW, Rodman JCS, Sugiyama O, Kohn DB, Lieberman JR. PMID: 35859364; PMCID: PMC9808795.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    23. The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions. J Allergy Clin Immunol. 2023 02; 151(2):547-555.e5. Dvorak CC, Haddad E, Heimall J, Dunn E, Cowan MJ, Pai SY, Kapoor N, Satter LF, Buckley RH, O'Reilly RJ, Chandra S, Bednarski JJ, Williams O, Rayes A, Moore TB, Ebens CL, Davila Saldana BJ, Petrovic A, Chellapandian D, Cuvelier GDE, Vander Lugt MT, Caywood EH, Chandrakasan S, Eissa H, Goldman FD, Shereck E, Aquino VM, Desantes KB, Madden LM, Miller HK, Yu L, Broglie L, Gillio A, Shah AJ, Knutsen AP, Andolina JP, Joshi AY, Szabolcs P, Kapadia M, Martinez CA, Parrot RE, Sullivan KE, Prockop SE, Abraham RS, Thakar MS, Leiding JW, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Puck JM. PMID: 36456360; PMCID: PMC9905305.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    24. The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions. J Allergy Clin Immunol. 2023 02; 151(2):539-546. Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB, Cowan MJ, Pai SY, Griffith LM, Cuvelier GDE, Eissa H, Shah AJ, O'Reilly RJ, Pulsipher MA, Wright NAM, Abraham RS, Satter LF, Notarangelo LD, Puck JM. PMID: 36456361; PMCID: PMC9905311.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCells
    25. Evaluation of clonal hematopoiesis in pediatric ADA-SCID gene therapy participants. Blood Adv. 2022 11 08; 6(21):5732-5736. White SL, Lee TD, Toy T, Carroll JE, Polsky L, Campo Fernandez B, Davila A, Kohn DB, Chang VY. PMID: 35914227; PMCID: PMC9619180.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCTClinical Trials
    26. Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential. Gene Ther. 2023 04; 30(3-4):369-376. Collon K, Bell JA, Gallo MC, Chang SW, Bougioukli S, Sugiyama O, Tassey J, Hollis R, Heckmann N, Oakes DA, Longjohn DB, Evseenko D, Kohn DB, Lieberman JR. PMID: 36216880; PMCID: PMC10086075.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimalsCells
    27. Diverse Approaches to Gene Therapy of Sickle Cell Disease. Annu Rev Med. 2023 01 27; 74:473-487. White SL, Hart K, Kohn DB. PMID: 36067800.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCells
    28. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC. Blood. 2022 08 18; 140(7):685-705. Cuvelier GDE, Logan BR, Prockop SE, Buckley RH, Kuo CY, Griffith LM, Liu X, Yip A, Hershfield MS, Ayoub PG, Moore TB, Dorsey MJ, O'Reilly RJ, Kapoor N, Pai SY, Kapadia M, Ebens CL, Forbes Satter LR, Burroughs LM, Petrovic A, Chellapandian D, Heimall J, Shyr DC, Rayes A, Bednarski JJ, Chandra S, Chandrakasan S, Gillio AP, Madden L, Quigg TC, Caywood EH, Dávila Saldaña BJ, DeSantes K, Eissa H, Goldman FD, Rozmus J, Shah AJ, Vander Lugt MT, Thakar MS, Parrott RE, Martinez C, Leiding JW, Torgerson TR, Pulsipher MA, Notarangelo LD, Cowan MJ, Dvorak CC, Haddad E, Puck JM, Kohn DB. PMID: 35671392; PMCID: PMC9389638.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCTClinical Trials
    29. Apheresis of Deceased Donors as a New Source of Mobilized Peripheral Blood Hematopoietic Stem Cells for Transplant Tolerance. Transplantation. 2023 02 01; 107(2):504-510. Sosa RA, Mone T, Naini BV, Kohn DB, Reed EF, Wheeler K, Campo-Fernandez B, Davila A, Chaffin DJ, DiNorcia J, Kaldas FM, Cohen A, Lum EL, Veale JL, Kogut NM. PMID: 35974436; PMCID: PMC9877104.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    30. Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation. J Allergy Clin Immunol. 2023 01; 151(1):260-271. Labrosse R, Boufaied I, Bourdin B, Gona S, Randolph HE, Logan BR, Bourbonnais S, Berthe C, Chan W, Buckley RH, Parrott RE, Cuvelier GDE, Kapoor N, Chandra S, Dávila Saldaña BJ, Eissa H, Goldman FD, Heimall J, O'Reilly R, Chaudhury S, Kolb EA, Shenoy S, Griffith LM, Pulsipher M, Kohn DB, Notarangelo LD, Pai SY, Cowan MJ, Dvorak CC, Haddad É, Puck JM, Barreiro LB, Decaluwe H. PMID: 35987350; PMCID: PMC9924130.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    31. Findings on Optical Coherence Tomography in Malignant Infantile Osteopetrosis. Ophthalmic Surg Lasers Imaging Retina. 2022 07; 53(7):398-402. Campagna GA, Chew L, Pettenkofer M, Nicoletti E, Schwartz JD, Choi G, Fernandes AO, Terrazas D, Kohn DB, Tsui I. PMID: 35858232.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    32. High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation. iScience. 2022 Jun 17; 25(6):104374. Magis W, DeWitt MA, Wyman SK, Vu JT, Heo SJ, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, Martin DIK. PMID: 35633935; PMCID: PMC9130532.
      View in: PubMed   Mentions: 19  
    33. Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy. J Clin Immunol. 2022 07; 42(5):1026-1035. Arnold DE, Chellapandian D, Parikh S, Mallhi K, Marsh RA, Heimall JR, Grossman D, Chitty-Lopez M, Murguia-Favela L, Gennery AR, Boulad F, Arbuckle E, Cowan MJ, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Kang EM, Malech HL, Leiding JW. PMID: 35445907; PMCID: PMC9022412.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    34. Previous Infection Combined with Vaccination Produces Neutralizing Antibodies with Potency against SARS-CoV-2 Variants. mBio. 2021 12 21; 12(6):e0265621. Ibarrondo FJ, Hofmann C, Ali A, Ayoub P, Kohn DB, Yang OO. PMID: 34872349; PMCID: PMC8649753.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCellsPHPublic Health
    35. Improved lentiviral vector titers from a multi-gene knockout packaging line. Mol Ther Oncolytics. 2021 Dec 17; 23:582-592. Han J, Tam K, Tam C, Hollis RP, Kohn DB. PMID: 34938858; PMCID: PMC8660686.
      View in: PubMed   Mentions: 5  
    36. Development of allogeneic HSC-engineered iNKT cells for off-the-shelf cancer immunotherapy. Cell Rep Med. 2021 11 16; 2(11):100449. Li YR, Zhou Y, Kim YJ, Zhu Y, Ma F, Yu J, Wang YC, Chen X, Li Z, Zeng S, Wang X, Lee D, Ku J, Tsao T, Hardoy C, Huang J, Cheng D, Montel-Hagen A, Seet CS, Crooks GM, Larson SM, Sasine JP, Wang X, Pellegrini M, Ribas A, Kohn DB, Witte O, Wang P, Yang L. PMID: 34841295; PMCID: PMC8607011.
      View in: PubMed   Mentions: 33     Fields:    Translation:HumansAnimalsCells
    37. Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency. Blood. 2021 10 14; 138(15):1304-1316. Reinhardt B, Habib O, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Terrazas D, Fernandez BC, De Oliveira S, Moore TB, Ikeda AK, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson C, Shupien S, Mishra S, Davila A, Mottahedeh J, Vitomirov A, Meng W, Rosenfeld AM, Roche AM, Hokama P, Reddy S, Everett J, Wang X, Luning Prak ET, Cornetta K, Hershfield MS, Sokolic R, De Ravin SS, Malech HL, Bushman FD, Candotti F, Kohn DB. PMID: 33974038; PMCID: PMC8525336.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCTClinical Trials
    38. Regional Gene Therapy with Transduced Human Cells: The Influence of "Cell Dose" on Bone Repair. Tissue Eng Part A. 2021 11; 27(21-22):1422-1433. Ihn H, Kang H, Iglesias B, Sugiyama O, Tang A, Hollis R, Bougioukli S, Skorka T, Park S, Longjohn D, Oakes DA, Kohn DB, Lieberman JR. PMID: 33882718; PMCID: PMC8827114.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansAnimals
    39. Evidence generation and reproducibility in cell and gene therapy research: A call to action. Mol Ther Methods Clin Dev. 2021 Sep 10; 22:11-14. Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, Goldstein LSB, Gottschalk S, Hamburg MA, Ingber DE, Kohn DB, Krainer AR, Maus MV, Marks P, Mummery CL, Pettigrew RI, Rutter JL, Teichmann SA, Terzic A, Urnov FD, Williams DA, Wolchok JD, Lawler M, Turtle CJ, Bauer G, Ioannidis JPA. PMID: 34377737; PMCID: PMC8322039.
      View in: PubMed   Mentions: 5  
    40. Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy. J Clin Immunol. 2021 10; 41(7):1597-1606. Baloh CH, Borkar SA, Chang KF, Yao J, Hershfield MS, Parikh SH, Kohn DB, Goodenow MM, Sleasman JW, Yin L. PMID: 34184208; PMCID: PMC9906566.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    41. Regional gene therapy for bone healing using a 3D printed scaffold in a rat femoral defect model. J Biomed Mater Res A. 2021 11; 109(11):2346-2356. Kang HP, Ihn H, Robertson DM, Chen X, Sugiyama O, Tang A, Hollis R, Skorka T, Longjohn D, Oakes D, Shah R, Kohn D, Jakus AE, Lieberman JR. PMID: 34018305.
      View in: PubMed   Mentions: 4     Fields:    Translation:Animals
    42. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 05 27; 384(21):2002-2013. Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. PMID: 33974366; PMCID: PMC8240285.
      View in: PubMed   Mentions: 61     Fields:    Translation:HumansCellsCTClinical Trials
    43. Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia. CRISPR J. 2021 04; 4(2):191-206. Gray DH, Villegas I, Long J, Santos J, Keir A, Abele A, Kuo CY, Kohn DB. PMID: 33876953; PMCID: PMC8336228.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCells
    44. Antiviral drug screen identifies DNA-damage response inhibitor as potent blocker of SARS-CoV-2 replication. Cell Rep. 2021 04 06; 35(1):108940. Garcia G, Sharma A, Ramaiah A, Sen C, Purkayastha A, Kohn DB, Parcells MS, Beck S, Kim H, Bakowski MA, Kirkpatrick MG, Riva L, Wolff KC, Han B, Yuen C, Ulmert D, Purbey PK, Scumpia P, Beutler N, Rogers TF, Chatterjee AK, Gabriel G, Bartenschlager R, Gomperts B, Svendsen CN, Betz UAK, Damoiseaux RD, Arumugaswami V. PMID: 33784499; PMCID: PMC7969873.
      View in: PubMed   Mentions: 52     Fields:    Translation:HumansAnimalsCells
    45. Gene Therapies for Primary Immune Deficiencies. Front Immunol. 2021; 12:648951. Kohn LA, Kohn DB. PMID: 33717203; PMCID: PMC7946985.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCells
    46. Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID. Mol Ther Methods Clin Dev. 2021 Mar 12; 20:765-778. Carbonaro-Sarracino DA, Chun K, Clark DN, Kaufman ML, Jin X, Wang X, Kohn DB. PMID: 33738330; PMCID: PMC7940710.
      View in: PubMed   Mentions: 1  
    47. Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers. J Clin Immunol. 2021 Feb; 41(2):498-500. Dorsey M, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Burroughs LM, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier G, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J. PMID: 33274413.
      View in: PubMed   Mentions:    Fields:    
    48. Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development. Hum Gene Ther. 2021 01; 32(1-2):31-42. Cannon P, Asokan A, Czechowicz A, Hammond P, Kohn DB, Lieber A, Malik P, Marks P, Porteus M, Verhoeyen E, Weissman D, Weissman I, Kiem HP. PMID: 33427035; PMCID: PMC8917879.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansCells
    49. Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells. Front Genome Ed. 2020; 2:601541. Benitez EK, Lomova Kaufman A, Cervantes L, Clark DN, Ayoub PG, Senadheera S, Osborne K, Sanchez JM, Crisostomo RV, Wang X, Reuven N, Shaul Y, Hollis RP, Romero Z, Kohn DB. PMID: 34713224; PMCID: PMC8525354.
      View in: PubMed   Mentions: 7  
    50. β-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production. Stem Cell Reports. 2021 01 12; 16(1):198-211. Han J, Tam K, Ma F, Tam C, Aleshe B, Wang X, Quintos JP, Morselli M, Pellegrini M, Hollis RP, Kohn DB. PMID: 33186538; PMCID: PMC7897704.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCells
    51. Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2020 Nov; 40(8):1211-1213. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Martinez C, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium. PMID: 32860171.
      View in: PubMed   Mentions:    Fields:    
    52. Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies. Sci Adv. 2020 10; 6(43). Yang P, Chou SJ, Li J, Hui W, Liu W, Sun N, Zhang RY, Zhu Y, Tsai ML, Lai HI, Smalley M, Zhang X, Chen J, Romero Z, Liu D, Ke Z, Zou C, Lee CF, Jonas SJ, Ban Q, Weiss PS, Kohn DB, Chen K, Chiou SH, Tseng HR. PMID: 33097539; PMCID: PMC7608838.
      View in: PubMed   Mentions: 16     Fields:    Translation:Animals
    53. Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers. J Clin Immunol. 2021 01; 41(1):38-50. Dorsey MJ, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier GDE, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J. PMID: 33006109; PMCID: PMC8388237.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansPHPublic Health
    54. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry. J Clin Immunol. 2020 11; 40(8):1124-1131. Kuo CY, Garabedian E, Puck J, Cowan MJ, Sullivan KE, Buckley RH, Cunningham-Rundles C, Marsh R, Candotti F, Kohn DB. PMID: 32880085; PMCID: PMC8216639.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansPHPublic Health
    55. Overview of the current status of gene therapy for primary immune deficiencies (PIDs). J Allergy Clin Immunol. 2020 08; 146(2):229-233. Kuo CY, Kohn DB. PMID: 32771134.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimals
    56. AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2020 11; 26(11):2061-2067. Bradford KL, Pearl M, Kohn DB, Weng P, Yadin O, Bowles V, De Oliveira SN, Moore TB. PMID: 32736008; PMCID: PMC8128374.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    57. Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy. Biol Blood Marrow Transplant. 2020 10; 26(10):1819-1827. Bradford KL, Liu S, Krajinovic M, Ansari M, Garabedian E, Tse J, Wang X, Shaw KL, Gaspar HB, Candotti F, Kohn DB. PMID: 32653625; PMCID: PMC7529956.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    58. Human CLEC9A antibodies deliver NY-ESO-1 antigen to CD141+ dendritic cells to activate naïve and memory NY-ESO-1-specific CD8+ T cells. J Immunother Cancer. 2020 07; 8(2). Masterman KA, Haigh OL, Tullett KM, Leal-Rojas IM, Walpole C, Pearson FE, Cebon J, Schmidt C, O'Brien L, Rosendahl N, Daraj G, Caminschi I, Gschweng EH, Hollis RP, Kohn DB, Lahoud MH, Radford KJ. PMID: 32737142; PMCID: PMC7394304.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansAnimalsCells
    59. Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia. Blood Adv. 2020 06 23; 4(12):2611-2616. Bosticardo M, Pala F, Calzoni E, Delmonte OM, Dobbs K, Gardner CL, Sacchetti N, Kawai T, Garabedian EK, Draper D, Bergerson JRE, DeRavin SS, Freeman AF, Güngör T, Hartog N, Holland SM, Kohn DB, Malech HL, Markert ML, Weinacht KG, Villa A, Seet CS, Montel-Hagen A, Crooks GM, Notarangelo LD. PMID: 32556283; PMCID: PMC7322962.
      View in: PubMed   Mentions: 41     Fields:    Translation:HumansCells
    60. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood. 2020 06 04; 135(23):2094-2105. Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing JJ, Edwards S, Dvorak CC, Chaudhury S, Prockop SE, Quinones R, Goldman FD, Quigg TC, Chandrakasan S, Smith AR, Parikh S, Dávila Saldaña BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez C, Chellapandian D, Shereck E, Miller HK, Kapoor N, Barnum JL, Chong H, Shyr DC, Chen K, Abu-Arja R, Shah AJ, Weinacht KG, Moore TB, Joshi A, DeSantes KB, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson T, Pulsipher MA, Haddad E, Sullivan KE, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings DJ, Cowan MJ. PMID: 32268350; PMCID: PMC7273831.
      View in: PubMed   Mentions: 42     Fields:    Translation:HumansCells
    61. Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:999-1013. Morgan RA, Ma F, Unti MJ, Brown D, Ayoub PG, Tam C, Lathrop L, Aleshe B, Kurita R, Nakamura Y, Senadheera S, Wong RL, Hollis RP, Pellegrini M, Kohn DB. PMID: 32426415; PMCID: PMC7225380.
      View in: PubMed   Mentions: 6  
    62. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey. Front Immunol. 2020; 11:239. Chan AY, Leiding JW, Liu X, Logan BR, Burroughs LM, Allenspach EJ, Skoda-Smith S, Uzel G, Notarangelo LD, Slatter M, Gennery AR, Smith AR, Pai SY, Jordan MB, Marsh RA, Cowan MJ, Dvorak CC, Craddock JA, Prockop SE, Chandrakasan S, Kapoor N, Buckley RH, Parikh S, Chellapandian D, Oshrine BR, Bednarski JJ, Cooper MA, Shenoy S, Davila Saldana BJ, Forbes LR, Martinez C, Haddad E, Shyr DC, Chen K, Sullivan KE, Heimall J, Wright N, Bhatia M, Cuvelier GDE, Goldman FD, Meyts I, Miller HK, Seidel MG, Vander Lugt MT, Bacchetta R, Weinacht KG, Andolina JR, Caywood E, Chong H, de la Morena MT, Aquino VM, Shereck E, Walter JE, Dorsey MJ, Seroogy CM, Griffith LM, Kohn DB, Puck JM, Pulsipher MA, Torgerson TR. PMID: 32153572; PMCID: PMC7046837.
      View in: PubMed   Mentions: 27     Fields:    Translation:HumansAnimalsCells
    63. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 02; 26(2):200-206. Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ, Net4CGD consortium. PMID: 31988463; PMCID: PMC7115833.
      View in: PubMed   Mentions: 92     Fields:    Translation:HumansCellsCTClinical Trials
    64. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Adv. 2019 12 10; 3(23):4002-4020. Farrell AT, Panepinto J, Desai AA, Kassim AA, Lebensburger J, Walters MC, Bauer DE, Blaylark RM, DiMichele DM, Gladwin MT, Green NS, Hassell K, Kato GJ, Klings ES, Kohn DB, Krishnamurti L, Little J, Makani J, Malik P, McGann PT, Minniti C, Morris CR, Odame I, Oneal PA, Setse R, Sharma P, Shenoy S. PMID: 31809537; PMCID: PMC6963248.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    65. Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice. Mol Ther Methods Clin Dev. 2020 Mar 13; 16:78-93. Carbonaro-Sarracino DA, Tarantal AF, Lee CCI, Kaufman ML, Wandro S, Jin X, Martinez M, Clark DN, Chun K, Koziol C, Hardee CL, Wang X, Kohn DB. PMID: 31871959; PMCID: PMC6909201.
      View in: PubMed   Mentions: 5  
    66. Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements. Mol Ther. 2020 01 08; 28(1):328-340. Morgan RA, Unti MJ, Aleshe B, Brown D, Osborne KS, Koziol C, Ayoub PG, Smith OB, O'Brien R, Tam C, Miyahira E, Ruiz M, Quintos JP, Senadheera S, Hollis RP, Kohn DB. PMID: 31628051; PMCID: PMC6953778.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansAnimalsCells
    67. Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer. Cell Stem Cell. 2019 10 03; 25(4):542-557.e9. Zhu Y, Smith DJ, Zhou Y, Li YR, Yu J, Lee D, Wang YC, Di Biase S, Wang X, Hardoy C, Ku J, Tsao T, Lin LJ, Pham AT, Moon H, McLaughlin J, Cheng D, Hollis RP, Campo-Fernandez B, Urbinati F, Wei L, Pang L, Rezek V, Berent-Maoz B, Macabali MH, Gjertson D, Wang X, Galic Z, Kitchen SG, An DS, Hu-Lieskovan S, Kaplan-Lefko PJ, De Oliveira SN, Seet CS, Larson SM, Forman SJ, Heath JR, Zack JA, Crooks GM, Radu CG, Ribas A, Kohn DB, Witte ON, Yang L. PMID: 31495780; PMCID: PMC7018522.
      View in: PubMed   Mentions: 29     Fields:    Translation:HumansAnimalsCells
    68. Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2019 10; 39(7):653-667. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium. PMID: 31376032; PMCID: PMC6754755.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCells
    69. Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates. Mol Ther. 2019 08 07; 27(8):1389-1406. Romero Z, Lomova A, Said S, Miggelbrink A, Kuo CY, Campo-Fernandez B, Hoban MD, Masiuk KE, Clark DN, Long J, Sanchez JM, Velez M, Miyahira E, Zhang R, Brown D, Wang X, Kurmangaliyev YZ, Hollis RP, Kohn DB. PMID: 31178391; PMCID: PMC6697408.
      View in: PubMed   Mentions: 50     Fields:    Translation:HumansCells
    70. PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector. Mol Ther Methods Clin Dev. 2019 Jun 14; 13:390-398. Masiuk KE, Zhang R, Osborne K, Hollis RP, Campo-Fernandez B, Kohn DB. PMID: 31024981; PMCID: PMC6477655.
      View in: PubMed   Mentions: 27  
    71. Correction to: Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization. Stem Cell Res Ther. 2019 Feb 12; 10(1):52. Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS. PMID: 30755264; PMCID: PMC6373121.
      View in: PubMed   Mentions:    Fields:    
    72. Chemistry, manufacturing and controls for gene modified hematopoietic stem cells. Cytotherapy. 2019 03; 21(3):358-366. Soni S, Kohn DB. PMID: 30745225.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCells
    73. Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017. Pediatrics. 2019 02; 143(2). Amatuni GS, Currier RJ, Church JA, Bishop T, Grimbacher E, Nguyen AA, Agarwal-Hashmi R, Aznar CP, Butte MJ, Cowan MJ, Dorsey MJ, Dvorak CC, Kapoor N, Kohn DB, Markert ML, Moore TB, Naides SJ, Sciortino S, Feuchtbaum L, Koupaei RA, Puck JM. PMID: 30683812; PMCID: PMC6361357.
      View in: PubMed   Mentions: 69     Fields:    Translation:HumansCellsPHPublic Health
    74. Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome. Cell Stem Cell. 2019 02 07; 24(2):309-317.e7. Masiuk KE, Laborada J, Roncarolo MG, Hollis RP, Kohn DB. PMID: 30639036.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansAnimalsCells
    75. Anti-human CD117 antibody-mediated bone marrow niche clearance in nonhuman primates and humanized NSG mice. Blood. 2019 05 09; 133(19):2104-2108. Kwon HS, Logan AC, Chhabra A, Pang WW, Czechowicz A, Tate K, Le A, Poyser J, Hollis R, Kelly BV, Kohn DB, Weissman IL, Prohaska SS, Shizuru JA. PMID: 30617195; PMCID: PMC6509543.
      View in: PubMed   Mentions: 43     Fields:    Translation:HumansAnimalsCells
    76. Gene therapy for blood diseases. Curr Opin Biotechnol. 2019 12; 60:39-45. Kohn DB. PMID: 30599357.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCells
    77. Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. Stem Cells. 2019 02; 37(2):284-294. Lomova A, Clark DN, Campo-Fernandez B, Flores-Bjurström C, Kaufman ML, Fitz-Gibbon S, Wang X, Miyahira EY, Brown D, DeWitt MA, Corn JE, Hollis RP, Romero Z, Kohn DB. PMID: 30372555; PMCID: PMC6368869.
      View in: PubMed   Mentions: 48     Fields:    Translation:HumansAnimalsCells
    78. IND-Enabling Studies for a Clinical Trial to Genetically Program a Persistent Cancer-Targeted Immune System. Clin Cancer Res. 2019 02 01; 25(3):1000-1011. Puig-Saus C, Parisi G, Garcia-Diaz A, Krystofinski PE, Sandoval S, Zhang R, Champhekar AS, McCabe J, Cheung-Lau GC, Truong NA, Vega-Crespo A, Komenan MDS, Pang J, Macabali MH, Saco JD, Goodwin JL, Bolon B, Seet CS, Montel-Hagen A, Crooks GM, Hollis RP, Campo-Fernandez B, Bischof D, Cornetta K, Gschweng EH, Adelson C, Nguyen A, Yang L, Witte ON, Baltimore D, Comin-Anduix B, Kohn DB, Wang X, Cabrera P, Kaplan-Lefko PJ, Berent-Maoz B, Ribas A. PMID: 30409823; PMCID: PMC6359988.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    79. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease. Mol Ther Methods Clin Dev. 2018 Dec 14; 11:167-179. Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F. PMID: 30533448; PMCID: PMC6276308.
      View in: PubMed   Mentions: 11  
    80. Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study. Hum Gene Ther. 2018 10; 29(10):1153-1166. Urbinati F, Campo Fernandez B, Masiuk KE, Poletti V, Hollis RP, Koziol C, Kaufman ML, Brown D, Mavilio F, Kohn DB. PMID: 30198339.
      View in: PubMed   Mentions: 21     Fields:    Translation:HumansAnimalsCells
    81. T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. Genome Med. 2018 09 28; 10(1):70. Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. PMID: 30261899; PMCID: PMC6161392.
      View in: PubMed   Mentions: 21     Fields:    Translation:HumansCells
    82. Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization. Gene Ther. 2018 10; 25(7):454-472. Hu P, Bi Y, Ma H, Suwanmanee T, Zeithaml B, Fry NJ, Kohn DB, Kafri T. PMID: 30190607; PMCID: PMC6478381.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    83. The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018). J Allergy Clin Immunol. 2019 01; 143(1):405-407. Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Logan B, Griffith LM, Kohn DB, Pai SY, Notarangelo L, Shearer W, Prockop S, Kapoor N, Heimall J, Chaudhury S, Shyr D, Chandra S, Cuvelier G, Moore T, Shenoy S, Goldman F, Smith AR, Sunkersett G, Vander Lugt M, Caywood E, Quigg T, Torgerson T, Chandrakasan S, Craddock J, Dávila Saldaña BJ, Gillio A, Shereck E, Aquino V, DeSantes K, Knutsen A, Thakar M, Yu L, Puck JM. PMID: 30193840; PMCID: PMC6322970.
      View in: PubMed   Mentions: 38     Fields:    Translation:HumansCTClinical Trials
    84. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. J Allergy Clin Immunol. 2019 03; 143(3):852-863. Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, Gaspar HB, Notarangelo LD, Grunebaum E. PMID: 30194989; PMCID: PMC6688493.
      View in: PubMed   Mentions: 52     Fields:    Translation:HumansAnimals
    85. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery. Blood. 2018 10 25; 132(17):1737-1749. Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai SY, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD. PMID: 30154114; PMCID: PMC6202916.
      View in: PubMed   Mentions: 82     Fields:    Translation:HumansCells
    86. Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome. Cell Rep. 2018 05 29; 23(9):2606-2616. Kuo CY, Long JD, Campo-Fernandez B, de Oliveira S, Cooper AR, Romero Z, Hoban MD, Joglekar AV, Lill GR, Kaufman ML, Fitz-Gibbon S, Wang X, Hollis RP, Kohn DB. PMID: 29847792; PMCID: PMC6181643.
      View in: PubMed   Mentions: 72     Fields:    Translation:HumansAnimalsCells
    87. B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation. Blood. 2018 06 28; 131(26):2967-2977. Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY. PMID: 29728406; PMCID: PMC6024640.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCellsCTClinical Trials
    88. Leukocyte adhesion deficiency-I: A comprehensive review of all published cases. J Allergy Clin Immunol Pract. 2018 Jul - Aug; 6(4):1418-1420.e10. Almarza Novoa E, Kasbekar S, Thrasher AJ, Kohn DB, Sevilla J, Nguyen T, Schwartz JD, Bueren JA. PMID: 29371071.
      View in: PubMed   Mentions: 40     Fields:    Translation:HumansCells
    89. Gene therapy comes of age. Science. 2018 01 12; 359(6372). Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. PMID: 29326244.
      View in: PubMed   Mentions: 415     Fields:    Translation:HumansAnimals
    90. Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells. Mol Ther. 2018 02 07; 26(2):468-479. Long J, Hoban MD, Cooper AR, Kaufman ML, Kuo CY, Campo-Fernandez B, Lumaquin D, Hollis RP, Wang X, Kohn DB, Romero Z. PMID: 29221806; PMCID: PMC5835144.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCells
    91. Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned. Cell Stem Cell. 2017 Nov 02; 21(5):574-590. Morgan RA, Gray D, Lomova A, Kohn DB. PMID: 29100011; PMCID: PMC6039108.
      View in: PubMed   Mentions: 103     Fields:    Translation:HumansCells
    92. Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study. Blood. 2017 12 21; 130(25):2718-2727. Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC. PMID: 29021228; PMCID: PMC5746165.
      View in: PubMed   Mentions: 96     Fields:    Translation:HumansPHPublic Health
    93. Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization. Stem Cell Res Ther. 2017 10 02; 8(1):217. Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS. PMID: 28969679; PMCID: PMC5625837.
      View in: PubMed   Mentions: 34     Fields:    Translation:HumansAnimalsCells
    94. Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy. Hematol Oncol Clin North Am. 2017 10; 31(5):721-735. Kohn DB. PMID: 28895843.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    95. Gene therapy: WAS (not) just for kids. Blood. 2017 09 14; 130(11):1278-1279. Kohn DB. PMID: 28912292.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansAnimals
    96. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations. J Clin Immunol. 2017 Oct; 37(7):626-637. Bradford KL, Moretti FA, Carbonaro-Sarracino DA, Gaspar HB, Kohn DB. PMID: 28842866.
      View in: PubMed   Mentions: 41     Fields:    Translation:HumansAnimals
    97. Gene Therapy. Hematol Oncol Clin North Am. 2017 10; 31(5):xiii-xiv. Bauer DE, Kohn DB. PMID: 28895857.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    98. Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease. Cytotherapy. 2017 09; 19(9):1096-1112. Urbinati F, Wherley J, Geiger S, Fernandez BC, Kaufman ML, Cooper A, Romero Z, Marchioni F, Reeves L, Read E, Nowicki B, Grassman E, Viswanathan S, Wang X, Hollis RP, Kohn DB. PMID: 28733131.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansAnimalsCells
    99. Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells. Mol Ther. 2017 09 06; 25(9):2163-2175. Masiuk KE, Brown D, Laborada J, Hollis RP, Urbinati F, Kohn DB. PMID: 28663101; PMCID: PMC5589063.
      View in: PubMed   Mentions: 19     Fields:    Translation:HumansAnimalsCells
    100. Generation of mature T cells from human hematopoietic stem and progenitor cells in artificial thymic organoids. Nat Methods. 2017 May; 14(5):521-530. Seet CS, He C, Bethune MT, Li S, Chick B, Gschweng EH, Zhu Y, Kim K, Kohn DB, Baltimore D, Crooks GM, Montel-Hagen A. PMID: 28369043; PMCID: PMC5426913.
      View in: PubMed   Mentions: 102     Fields:    Translation:HumansCells
    101. Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients. Blood. 2017 05 11; 129(19):2624-2635. Cooper AR, Lill GR, Shaw K, Carbonaro-Sarracino DA, Davila A, Sokolic R, Candotti F, Pellegrini M, Kohn DB. PMID: 28351939; PMCID: PMC5428461.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCellsCTClinical Trials
    102. Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency. J Clin Invest. 2017 May 01; 127(5):1689-1699. Shaw KL, Garabedian E, Mishra S, Barman P, Davila A, Carbonaro D, Shupien S, Silvin C, Geiger S, Nowicki B, Smogorzewska EM, Brown B, Wang X, de Oliveira S, Choi Y, Ikeda A, Terrazas D, Fu PY, Yu A, Fernandez BC, Cooper AR, Engel B, Podsakoff G, Balamurugan A, Anderson S, Muul L, Jagadeesh GJ, Kapoor N, Tse J, Moore TB, Purdy K, Rishi R, Mohan K, Skoda-Smith S, Buchbinder D, Abraham RS, Scharenberg A, Yang OO, Cornetta K, Gjertson D, Hershfield M, Sokolic R, Candotti F, Kohn DB. PMID: 28346229; PMCID: PMC5409097.
      View in: PubMed   Mentions: 43     Fields:    Translation:HumansCellsCTClinical Trials
    103. New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing. J Allergy Clin Immunol. 2017 Mar; 139(3):726-732. Kohn DB, Kuo CY. PMID: 28270364; PMCID: PMC5911283.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansAnimals
    104. How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID). J Clin Immunol. 2017 May; 37(4):351-356. Kohn DB, Gaspar HB. PMID: 28194615.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansPHPublic Health
    105. Domain-swapped T cell receptors improve the safety of TCR gene therapy. Elife. 2016 11 08; 5. Bethune MT, Gee MH, Bunse M, Lee MS, Gschweng EH, Pagadala MS, Zhou J, Cheng D, Heath JR, Kohn DB, Kuhns MS, Uckert W, Baltimore D. PMID: 27823582; PMCID: PMC5101000.
      View in: PubMed   Mentions: 29     Fields:    Translation:AnimalsCells
    106. Propagating Humanized BLT Mice for the Study of Human Immunology and Immunotherapy. Stem Cells Dev. 2016 12 15; 25(24):1863-1873. Smith DJ, Lin LJ, Moon H, Pham AT, Wang X, Liu S, Ji S, Rezek V, Shimizu S, Ruiz M, Lam J, Janzen DM, Memarzadeh S, Kohn DB, Zack JA, Kitchen SG, An DS, Yang L. PMID: 27608727; PMCID: PMC5165681.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansAnimalsCells
    107. Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. Sci Transl Med. 2016 10 12; 8(360):360ra134. DeWitt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, Heo SJ, Mitros T, Muñoz DP, Boffelli D, Kohn DB, Walters MC, Carroll D, Martin DI, Corn JE. PMID: 27733558; PMCID: PMC5500303.
      View in: PubMed   Mentions: 211     Fields:    Translation:HumansAnimalsCells
    108. Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency. Hum Gene Ther. 2017 01; 28(1):112-124. Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta K, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, Puck JM, Cowan MJ. PMID: 27611239; PMCID: PMC5278830.
      View in: PubMed   Mentions: 21     Fields:    Translation:HumansAnimalsCells
    109. CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells. Mol Ther. 2016 Sep; 24(9):1561-9. Hoban MD, Lumaquin D, Kuo CY, Romero Z, Long J, Ho M, Young CS, Mojadidi M, Fitz-Gibbon S, Cooper AR, Lill GR, Urbinati F, Campo-Fernandez B, Bjurstrom CF, Pellegrini M, Hollis RP, Kohn DB. PMID: 27406980; PMCID: PMC5113113.
      View in: PubMed   Mentions: 85     Fields:    Translation:HumansCells
    110. Analyzing CRISPR genome-editing experiments with CRISPResso. Nat Biotechnol. 2016 07 12; 34(7):695-7. Pinello L, Canver MC, Hoban MD, Orkin SH, Kohn DB, Bauer DE, Yuan GC. PMID: 27404874; PMCID: PMC5242601.
      View in: PubMed   Mentions: 239     Fields:    Translation:HumansAnimalsCells
    111. Gene Therapy for the Treatment of Primary Immune Deficiencies. Curr Allergy Asthma Rep. 2016 May; 16(5):39. Kuo CY, Kohn DB. PMID: 27056559; PMCID: PMC5909980.
      View in: PubMed   Mentions: 22     Fields:    Translation:HumansAnimalsCells
    112. Primary Immune Deficiency Treatment Consortium (PIDTC) update. J Allergy Clin Immunol. 2016 08; 138(2):375-85. Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Shearer WT, Burroughs LM, Torgerson TR, Decaluwe H, Haddad E, workshop participants. PMID: 27262745; PMCID: PMC4986691.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansAnimals
    113. Ethical and regulatory aspects of genome editing. Blood. 2016 05 26; 127(21):2553-60. Kohn DB, Porteus MH, Scharenberg AM. PMID: 27053531.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansAnimals
    114. Genetic Tagging During Human Mesoderm Differentiation Reveals Tripotent Lateral Plate Mesodermal Progenitors. Stem Cells. 2016 05; 34(5):1239-50. Chin CJ, Cooper AR, Lill GR, Evseenko D, Zhu Y, He CB, Casero D, Pellegrini M, Kohn DB, Crooks GM. PMID: 26934332; PMCID: PMC5052131.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansAnimalsCells
    115. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Cell Stem Cell. 2016 Apr 07; 18(4):533-40. Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, Pyle AD. PMID: 26877224; PMCID: PMC4826286.
      View in: PubMed   Mentions: 208     Fields:    Translation:HumansAnimalsCells
    116. Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells. Curr Protoc Stem Cell Biol. 2016 Feb 03; 36:5B.4.1-5B.4.10. Hoban MD, Romero Z, Cost GJ, Mendel M, Holmes M, Kohn DB. PMID: 26840227.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansAnimalsCells
    117. Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases. Mol Ther Nucleic Acids. 2016; 5:e351. Bjurström CF, Mojadidi M, Phillips J, Kuo C, Lai S, Lill GR, Cooper A, Kaufman M, Urbinati F, Wang X, Hollis RP, Kohn DB. PMID: 28131278; PMCID: PMC5023398.
      View in: PubMed   Mentions: 32  
    118. Impulse oscillometry identifies peripheral airway dysfunction in children with adenosine deaminase deficiency. Orphanet J Rare Dis. 2015 Dec 18; 10:159. Komarow HD, Sokolic R, Hershfield MS, Kohn DB, Young M, Metcalfe DD, Candotti F. PMID: 26682746; PMCID: PMC4683718.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    119. A Journey in the Development of Gene Therapy for Inherited Disorders of the Bone Marrow. Hum Gene Ther Clin Dev. 2015 Dec; 26(4):203-7. Kohn D, Wilson JM. PMID: 26684443.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    120. Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection. Am J Hematol. 2015 Nov; 90(11):1021-6. Abdel-Azim H, Mahadeo KM, Zhao Q, Khazal S, Kohn DB, Crooks GM, Shah AJ, Kapoor N. PMID: 26242764.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    121. Gene therapy outpaces haplo for SCID-X1. Blood. 2015 Jun 04; 125(23):3521-2. Kohn DB. PMID: 26045591.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCells
    122. Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy. Stem Cells. 2015 May; 33(5):1532-42. Baldwin K, Urbinati F, Romero Z, Campo-Fernandez B, Kaufman ML, Cooper AR, Masiuk K, Hollis RP, Kohn DB. PMID: 25588820; PMCID: PMC5074681.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansAnimalsCells
    123. Genome editing technologies: defining a path to clinic. Mol Ther. 2015 May; 23(5):796-806. Corrigan-Curay J, O'Reilly M, Kohn DB, Cannon PM, Bao G, Bushman FD, Carroll D, Cathomen T, Joung JK, Roth D, Sadelain M, Scharenberg AM, von Kalle C, Zhang F, Jambou R, Rosenthal E, Hassani M, Singh A, Porteus MH. PMID: 25943494; PMCID: PMC4427885.
      View in: PubMed   Mentions: 45     Fields:    Translation:HumansAnimalsCells
    124. The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells. Mol Ther Methods Clin Dev. 2015; 2:15012. Romero Z, Campo-Fernandez B, Wherley J, Kaufman ML, Urbinati F, Cooper AR, Hoban MD, Baldwin KM, Lumaquin D, Wang X, Senadheera S, Hollis RP, Kohn DB. PMID: 26029723; PMCID: PMC4445009.
      View in: PubMed   Mentions: 10  
    125. Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. Blood. 2015 Apr 23; 125(17):2597-604. Hoban MD, Cost GJ, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu PQ, Paschon DE, Zhang L, Rebar EJ, Wilber A, Wang X, Gregory PD, Holmes MC, Reik A, Hollis RP, Kohn DB. PMID: 25733580; PMCID: PMC4408287.
      View in: PubMed   Mentions: 172     Fields:    Translation:HumansAnimalsCells
    126. Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells. Exp Hematol. 2015 May; 43(5):346-351. Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A. PMID: 25681747; PMCID: PMC4428920.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansCells
    127. Putative immunogenicity expression profiling using human pluripotent stem cells and derivatives. Stem Cells Transl Med. 2015 Feb; 4(2):136-45. Awe JP, Gschweng EH, Vega-Crespo A, Voutila J, Williamson MH, Truong B, Kohn DB, Kasahara N, Byrne JA. PMID: 25575527; PMCID: PMC4303355.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimalsCells
    128. Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter. Nucleic Acids Res. 2015 Jan; 43(1):682-90. Cooper AR, Lill GR, Gschweng EH, Kohn DB. PMID: 25520191; PMCID: PMC4288199.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCells
    129. Eliminating SCID row: new approaches to SCID. Hematology Am Soc Hematol Educ Program. 2014 Dec 05; 2014(1):475-80. Kohn DB. PMID: 25696897.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansPHPublic Health
    130. A reduced-toxicity regimen is associated with durable engraftment and clinical cure of nonmalignant genetic diseases among children undergoing blood and marrow transplantation with an HLA-matched related donor. Biol Blood Marrow Transplant. 2015 Mar; 21(3):440-4. Mahadeo KM, Weinberg KI, Abdel-Azim H, Miklos DB, Killen R, Kohn D, Crooks GM, Shah AJ, Kharbanda S, Agarwal R, Kapoor N. PMID: 25459642.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCTClinical Trials
    131. A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. PMID: 25295500; PMCID: PMC4274995.
      View in: PubMed   Mentions: 160     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
    132. T-cell immunotherapy: looking forward. Mol Ther. 2014 Sep; 22(9):1564-74. Corrigan-Curay J, Kiem HP, Baltimore D, O'Reilly M, Brentjens RJ, Cooper L, Forman S, Gottschalk S, Greenberg P, Junghans R, Heslop H, Jensen M, Mackall C, June C, Press O, Powell D, Ribas A, Rosenberg S, Sadelain M, Till B, Patterson AP, Jambou RC, Rosenthal E, Gargiulo L, Montgomery M, Kohn DB. PMID: 25186558; PMCID: PMC4435492.
      View in: PubMed   Mentions: 31     Fields:    Translation:HumansCells
    133. Envelope, please. And the award goes to…. Blood. 2014 Aug 21; 124(8):1203-4. Kohn DB, Hollis RP. PMID: 25147372; PMCID: PMC4141502.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansAnimalsCells
    134. Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States. JAMA. 2014 Aug 20; 312(7):729-38. Kwan A, Abraham RS, Currier R, Brower A, Andruszewski K, Abbott JK, Baker M, Ballow M, Bartoshesky LE, Bonilla FA, Brokopp C, Brooks E, Caggana M, Celestin J, Church JA, Comeau AM, Connelly JA, Cowan MJ, Cunningham-Rundles C, Dasu T, Dave N, De La Morena MT, Duffner U, Fong CT, Forbes L, Freedenberg D, Gelfand EW, Hale JE, Hanson IC, Hay BN, Hu D, Infante A, Johnson D, Kapoor N, Kay DM, Kohn DB, Lee R, Lehman H, Lin Z, Lorey F, Abdel-Mageed A, Manning A, McGhee S, Moore TB, Naides SJ, Notarangelo LD, Orange JS, Pai SY, Porteus M, Rodriguez R, Romberg N, Routes J, Ruehle M, Rubenstein A, Saavedra-Matiz CA, Scott G, Scott PM, Secord E, Seroogy C, Shearer WT, Siegel S, Silvers SK, Stiehm ER, Sugerman RW, Sullivan JL, Tanksley S, Tierce ML, Verbsky J, Vogel B, Walker R, Walkovich K, Walter JE, Wasserman RL, Watson MS, Weinberg GA, Weiner LB, Wood H, Yates AB, Puck JM, Bonagura VR. PMID: 25138334; PMCID: PMC4492158.
      View in: PubMed   Mentions: 250     Fields:    Translation:HumansCellsPHPublic Health
    135. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31; 371(5):434-46. Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ. PMID: 25075835; PMCID: PMC4183064.
      View in: PubMed   Mentions: 269     Fields:    Translation:HumansCells
    136. HSV-sr39TK positron emission tomography and suicide gene elimination of human hematopoietic stem cells and their progeny in humanized mice. Cancer Res. 2014 Sep 15; 74(18):5173-83. Gschweng EH, McCracken MN, Kaufman ML, Ho M, Hollis RP, Wang X, Saini N, Koya RC, Chodon T, Ribas A, Witte ON, Kohn DB. PMID: 25038231; PMCID: PMC4167467.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansAnimalsCells
    137. Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys. Mol Ther. 2014 Oct; 22(10):1803-16. Carbonaro Sarracino D, Tarantal AF, Lee CC, Martinez M, Jin X, Wang X, Hardee CL, Geiger S, Kahl CA, Kohn DB. PMID: 24925206; PMCID: PMC4428412.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    138. Erythropoiesis from human embryonic stem cells through erythropoietin-independent AKT signaling. Stem Cells. 2014 Jun; 32(6):1503-14. Kim WS, Zhu Y, Deng Q, Chin CJ, He CB, Grieco AJ, Dravid GG, Parekh C, Hollis RP, Lane TF, Bouhassira EE, Kohn DB, Crooks GM. PMID: 24677652; PMCID: PMC4037366.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansCells
    139. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Hum Gene Ther. 2014 Jun; 25(6):488-97. O'Reilly M, Federoff HJ, Fong Y, Kohn DB, Patterson AP, Ahmed N, Asokan A, Boye SE, Crystal RG, De Oliveira S, Gargiulo L, Harper SQ, Ikeda Y, Jambou R, Montgomery M, Prograis L, Rosenthal E, Sterman DH, Vandenberghe LH, Zoloth L, Abedi M, Adair J, Adusumilli PS, Goins WF, Gray J, Monahan P, Popplewell L, Sena-Esteves M, Tannous B, Weber T, Wierda W, Gopal-Srivastava R, McDonald CL, Rosenblum D, Corrigan-Curay J. PMID: 24773122; PMCID: PMC4064731.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansAnimals
    140. Human lymphoid development in the absence of common γ-chain receptor signaling. J Immunol. 2014 Jun 01; 192(11):5050-8. Kohn LA, Seet CS, Scholes J, Codrea F, Chan R, Zaidi-Merchant S, Zhu Y, De Oliveira S, Kapoor N, Shah A, Abdel-Azim H, Kohn DB, Crooks GM. PMID: 24771849; PMCID: PMC4052377.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
    141. Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors. Hum Gene Ther. 2014 Jul; 25(7):599-608. Joglekar AV, Stein L, Ho M, Hoban MD, Hollis RP, Kohn DB. PMID: 24568341; PMCID: PMC4098962.
      View in: PubMed   Mentions: 11     Fields:    Translation:HumansCells
    142. HIV eradication--from Berlin to Boston. Nat Biotechnol. 2014 Apr; 32(4):315-6. Cannon PM, Kohn DB, Kiem HP. PMID: 24714473.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCells
    143. Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma. Clin Cancer Res. 2014 May 01; 20(9):2457-65. Chodon T, Comin-Anduix B, Chmielowski B, Koya RC, Wu Z, Auerbach M, Ng C, Avramis E, Seja E, Villanueva A, McCannel TA, Ishiyama A, Czernin J, Radu CG, Wang X, Gjertson DW, Cochran AJ, Cornetta K, Wong DJ, Kaplan-Lefko P, Hamid O, Samlowski W, Cohen PA, Daniels GA, Mukherji B, Yang L, Zack JA, Kohn DB, Heath JR, Glaspy JA, Witte ON, Baltimore D, Economou JS, Ribas A. PMID: 24634374; PMCID: PMC4070853.
      View in: PubMed   Mentions: 113     Fields:    Translation:HumansCellsPHPublic HealthCTClinical Trials
    144. Hematopoietic stem cells for cancer immunotherapy. Immunol Rev. 2014 Jan; 257(1):237-49. Gschweng E, De Oliveira S, Kohn DB. PMID: 24329801; PMCID: PMC3901057.
      View in: PubMed   Mentions: 38     Fields:    Translation:HumansAnimalsCells
    145. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol. 2014 Apr; 133(4):1092-8. Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ. PMID: 24290292; PMCID: PMC3972266.
      View in: PubMed   Mentions: 139     Fields:    Translation:Humans
    146. Prostate cancer originating in basal cells progresses to adenocarcinoma propagated by luminal-like cells. Proc Natl Acad Sci U S A. 2013 Dec 10; 110(50):20111-6. Stoyanova T, Cooper AR, Drake JM, Liu X, Armstrong AJ, Pienta KJ, Zhang H, Kohn DB, Huang J, Witte ON, Goldstein AS. PMID: 24282295; PMCID: PMC3864278.
      View in: PubMed   Mentions: 99     Fields:    Translation:HumansCells
    147. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther. 2014 Mar; 22(3):607-622. Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. PMID: 24256635; PMCID: PMC3944341.
      View in: PubMed   Mentions: 42     Fields:    Translation:HumansAnimalsCells
    148. Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014 Feb; 133(2):335-47. Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC, workshop participants. PMID: 24139498; PMCID: PMC3960312.
      View in: PubMed   Mentions: 22     Fields:    Translation:HumansPHPublic Health
    149. Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. Hum Gene Ther. 2013 Oct; 24(10):824-39. De Oliveira SN, Ryan C, Giannoni F, Hardee CL, Tremcinska I, Katebian B, Wherley J, Sahaghian A, Tu A, Grogan T, Elashoff D, Cooper LJ, Hollis RP, Kohn DB. PMID: 23978226; PMCID: PMC3787487.
      View in: PubMed   Mentions: 39     Fields:    Translation:HumansAnimalsCells
    150. Generation and characterization of transgene-free human induced pluripotent stem cells and conversion to putative clinical-grade status. Stem Cell Res Ther. 2013 Jul 26; 4(4):87. Awe JP, Lee PC, Ramathal C, Vega-Crespo A, Durruthy-Durruthy J, Cooper A, Karumbayaram S, Lowry WE, Clark AT, Zack JA, Sebastiano V, Kohn DB, Pyle AD, Martin MG, Lipshutz GS, Phelps PE, Pera RA, Byrne JA. PMID: 23890092; PMCID: PMC3854769.
      View in: PubMed   Mentions: 26     Fields:    Translation:HumansAnimalsCells
    151. Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Mol Ther. 2013 Sep; 21(9):1705-17. Joglekar AV, Hollis RP, Kuftinec G, Senadheera S, Chan R, Kohn DB. PMID: 23857176; PMCID: PMC3776625.
      View in: PubMed   Mentions: 39     Fields:    Translation:HumansCells
    152. The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol. 2013 Oct; 33(7):1156-64. Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Shearer WT, O'Reilly RJ, Fleisher TA, Pai SY, Hanson IC, Pulsipher MA, Fuleihan R, Filipovich A, Goldman F, Kapoor N, Small T, Smith A, Chan KW, Cuvelier G, Heimall J, Knutsen A, Loechelt B, Moore T, Buckley RH. PMID: 23818196; PMCID: PMC3784642.
      View in: PubMed   Mentions: 61     Fields:    Translation:Humans
    153. Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years. J Allergy Clin Immunol. 2013 Jul; 132(1):140-50. Kwan A, Church JA, Cowan MJ, Agarwal R, Kapoor N, Kohn DB, Lewis DB, McGhee SA, Moore TB, Stiehm ER, Porteus M, Aznar CP, Currier R, Lorey F, Puck JM. PMID: 23810098; PMCID: PMC3759317.
      View in: PubMed   Mentions: 88     Fields:    Translation:HumansCellsPHPublic Health
    154. β-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest. 2013 Jul 01. Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, Kempert P, Shupien S, Abdel-Azim H, Walters MC, Meiselman HJ, Wenby RB, Gruber T, Marder V, Coates TD, Kohn DB. PMID: 23863630; PMCID: PMC4011030.
      View in: PubMed   Mentions: 62     Fields:    
    155. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther. 2013 Apr; 24(4):355-62. O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, Montgomery M, Rosenthal E, Samulski RJ, Skarlatos SI, Sorrentino B, Wilson JM, Xie Y, Corrigan-Curay J. PMID: 23517518; PMCID: PMC3631014.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    156. Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells. Mol Ther. 2013 May; 21(5):1044-54. Giannoni F, Hardee CL, Wherley J, Gschweng E, Senadheera S, Kaufman ML, Chan R, Bahner I, Gersuk V, Wang X, Gjertson D, Baltimore D, Witte ON, Economou JS, Ribas A, Kohn DB. PMID: 23380815; PMCID: PMC3666644.
      View in: PubMed   Mentions: 38     Fields:    Translation:HumansAnimalsCells
    157. A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors. J Transl Med. 2013 Jan 29; 11:23. De Oliveira SN, Wang J, Ryan C, Morrison SL, Kohn DB, Hollis RP. PMID: 23360526; PMCID: PMC3623748.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansAnimalsCells
    158. Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene. Proc Natl Acad Sci U S A. 2013 Jan 29; 110(5):1857-62. McCracken MN, Gschweng EH, Nair-Gill E, McLaughlin J, Cooper AR, Riedinger M, Cheng D, Nosala C, Kohn DB, Witte ON. PMID: 23319634; PMCID: PMC3562822.
      View in: PubMed   Mentions: 24     Fields:    Translation:AnimalsCells
    159. Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Mol Ther. 2013 Feb; 21(2):433-44. Harrison F, Yeagy BA, Rocca CJ, Kohn DB, Salomon DR, Cherqui S. PMID: 23089735; PMCID: PMC3594011.
      View in: PubMed   Mentions: 43     Fields:    Translation:AnimalsCells
    160. Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells. Biol Blood Marrow Transplant. 2013 Jan; 19(1 Suppl):S64-9. Kohn DB, Pai SY, Sadelain M. PMID: 23032601.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansAnimalsCells
    161. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood. 2012 Nov 01; 120(18):3635-46. Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, Schurman SH, Garabedian E, Kesserwan C, Jagadeesh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, Crooks GM, Kapoor N, Shah A, Abdel-Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland JA, Engel BC, Podsakoff GM, Hershfield MS, Blaese RM, Parkman R, Kohn DB. PMID: 22968453; PMCID: PMC3488882.
      View in: PubMed   Mentions: 111     Fields:    Translation:HumansCellsCTClinical Trials
    162. Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction. Blood. 2012 Nov 01; 120(18):3677-87. Carbonaro DA, Jin X, Wang X, Yu XJ, Rozengurt N, Kaufman ML, Wang X, Gjertson D, Zhou Y, Blackburn MR, Kohn DB. PMID: 22833548; PMCID: PMC3488883.
      View in: PubMed   Mentions: 19     Fields:    Translation:AnimalsCells
    163. Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther. 2012 Jun; 20(6):1084-94. Corrigan-Curay J, Cohen-Haguenauer O, O'Reilly M, Ross SR, Fan H, Rosenberg N, Somia N, King N, Friedmann T, Dunbar C, Aiuti A, Naldini L, Baum C, von Kalle C, Kiem HP, Montini E, Bushman F, Sorrentino BP, Carrondo M, Malech H, Gahrton G, Shapiro R, Wolff L, Rosenthal E, Jambou R, Zaia J, Kohn DB. PMID: 22652996; PMCID: PMC3369284.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCells
    164. Novel pathways to erythropoiesis induced by dimerization of intracellular C-Mpl in human hematopoietic progenitors. Stem Cells. 2012 Apr; 30(4):697-708. Parekh C, Sahaghian A, Kim W, Scholes J, Ge S, Zhu Y, Asgharzadeh S, Hollis R, Kohn D, Ji L, Malvar J, Wang X, Crooks G. PMID: 22290824; PMCID: PMC3973153.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    165. Nonmyeloablative conditioning regimen to increase engraftment of gene-modified hematopoietic stem cells in young rhesus monkeys. Mol Ther. 2012 May; 20(5):1033-45. Tarantal AF, Giannoni F, Lee CC, Wherley J, Sumiyoshi T, Martinez M, Kahl CA, Elashoff D, Louie SG, Kohn DB. PMID: 22294147; PMCID: PMC3345994.
      View in: PubMed   Mentions: 20     Fields:    Translation:AnimalsCells
    166. From skin biopsy to neurons through a pluripotent intermediate under Good Manufacturing Practice protocols. Stem Cells Transl Med. 2012 Jan; 1(1):36-43. Karumbayaram S, Lee P, Azghadi SF, Cooper AR, Patterson M, Kohn DB, Pyle A, Clark A, Byrne J, Zack JA, Plath K, Lowry WE. PMID: 23197638; PMCID: PMC3727693.
      View in: PubMed   Mentions: 29     Fields:    Translation:HumansAnimalsCells
    167. Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proc Natl Acad Sci U S A. 2011 Dec 20; 108(51):E1408-16. Vatakis DN, Koya RC, Nixon CC, Wei L, Kim SG, Avancena P, Bristol G, Baltimore D, Kohn DB, Ribas A, Radu CG, Galic Z, Zack JA. PMID: 22123951; PMCID: PMC3251070.
      View in: PubMed   Mentions: 69     Fields:    Translation:HumansAnimalsCells
    168. A tale of two SCIDs. Sci Transl Med. 2011 Aug 24; 3(97):97ps36. Shaw KL, Kohn DB. PMID: 21865536.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    169. Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. J Virol Methods. 2011 Oct; 177(1):1-9. Cooper AR, Patel S, Senadheera S, Plath K, Kohn DB, Hollis RP. PMID: 21784103; PMCID: PMC4276401.
      View in: PubMed   Mentions: 39     Fields:    Translation:HumansAnimalsCells
    170. Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood. 2011 Sep 08; 118(10):2688-94. Sokolic R, Maric I, Kesserwan C, Garabedian E, Hanson IC, Dodds M, Buckley R, Issekutz AC, Kamani N, Shaw K, Tan B, Bali P, Hershfield MS, Kohn DB, Wayne AS, Candotti F. PMID: 21725047; PMCID: PMC3172788.
      View in: PubMed   Mentions: 16     Fields:    Translation:Humans
    171. CARs on track in the clinic. Mol Ther. 2011 Mar; 19(3):432-8. Kohn DB, Dotti G, Brentjens R, Savoldo B, Jensen M, Cooper LJ, June CH, Rosenberg S, Sadelain M, Heslop HE. PMID: 21358705; PMCID: PMC3048197.
      View in: PubMed   Mentions: 65     Fields:    Translation:Humans
    172. Guidance for developing phase II cell therapy trial proposals for consideration by the Blood and Marrow Transplant Clinical Trials Network. Biol Blood Marrow Transplant. 2011 Feb; 17(2):192-6. Horwitz EM, Horowitz MM, DiFronzo NL, Kohn DB, Heslop HE, Blood and Marrow Transplant Clinical Trials Network, State of the Science Cell and Gene Therapy Comm. PMID: 21182973; PMCID: PMC3387575.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    173. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol. 2010 Aug; 28(8):839-47. Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM. PMID: 20601939; PMCID: PMC3080757.
      View in: PubMed   Mentions: 360     Fields:    Translation:HumansAnimalsCells
    174. Regulated expansion of human pancreatic beta-cells. Mol Ther. 2010 Jul; 18(7):1389-96. Pais E, Park J, Alexy T, Nikolian V, Ge S, Shaw K, Senadheera S, Hardee CL, Skelton D, Hollis R, Crooks GM, Kohn DB. PMID: 20389286; PMCID: PMC2911252.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimalsCells
    175. Neurocognitive function of patients with severe combined immunodeficiency. Immunol Allergy Clin North Am. 2010 Feb; 30(1):143-51. Shah AJ, Kohn DB. PMID: 20113891.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    176. Update on gene therapy for immunodeficiencies. Clin Immunol. 2010 May; 135(2):247-54. Kohn DB. PMID: 20071242; PMCID: PMC2856741.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCells
    177. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. J Allergy Clin Immunol. 2009 Dec; 124(6):1152-60.e12. Griffith LM, Cowan MJ, Notarangelo LD, Puck JM, Buckley RH, Candotti F, Conley ME, Fleisher TA, Gaspar HB, Kohn DB, Ochs HD, O'Reilly RJ, Rizzo JD, Roifman CM, Small TN, Shearer WT, Workshop Participants. PMID: 20004776; PMCID: PMC2831471.
      View in: PubMed   Mentions: 52     Fields:    Translation:Humans
    178. Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system. Hum Gene Ther. 2009 Dec; 20(12):1607-26. Sumiyoshi T, Holt NG, Hollis RP, Ge S, Cannon PM, Crooks GM, Kohn DB. PMID: 19689196; PMCID: PMC4094131.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansAnimalsCells
    179. Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency. Hum Gene Ther. 2009 Apr; 20(4):337-49. Sondergaard CS, Haldrup C, Beer C, Andersen B, Kohn DB, Pedersen L. PMID: 19281432.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansAnimalsCells
    180. Gene therapy fulfilling its promise. N Engl J Med. 2009 Jan 29; 360(5):518-21. Kohn DB, Candotti F. PMID: 19179320.
      View in: PubMed   Mentions: 24     Fields:    Translation:HumansCells
    181. Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2009 Jan; 52(1):139-42. Shah AJ, Kapoor N, Cooper RM, Crooks GM, Lenarsky C, Abdel-Azim H, Wu SQ, Wilson K, Weinberg KI, Parkman R, Kohn DB. PMID: 18819128; PMCID: PMC2592085.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    182. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J Allergy Clin Immunol. 2008 Dec; 122(6):1087-96. Griffith LM, Cowan MJ, Kohn DB, Notarangelo LD, Puck JM, Schultz KR, Buckley RH, Eapen M, Kamani NR, O'Reilly RJ, Parkman R, Roifman CM, Sullivan KE, Filipovich AH, Fleisher TA, Shearer WT. PMID: 18992926; PMCID: PMC3357108.
      View in: PubMed   Mentions: 34     Fields:    Translation:Humans
    183. Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT). J Clin Immunol. 2009 Mar; 29(2):231-7. Lin M, Epport K, Azen C, Parkman R, Kohn DB, Shah AJ. PMID: 18807155; PMCID: PMC7102058.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    184. Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency. Pediatr Transplant. 2009 Mar; 13(2):244-50. Gruber TA, Shah AJ, Hernandez M, Crooks GM, Abdel-Azim H, Gupta S, McKnight S, White D, Kapoor N, Kohn DB. PMID: 18822103.
      View in: PubMed   Mentions: 11     Fields:    Translation:HumansCells
    185. CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma model. J Immunol. 2008 Jul 15; 181(2):1063-70. Chhabra A, Yang L, Wang P, Comin-Anduix B, Das R, Chakraborty NG, Ray S, Mehrotra S, Yang H, Hardee CL, Hollis R, Dorsky DI, Koya R, Kohn DB, Ribas A, Economou JS, Baltimore D, Mukherji B. PMID: 18606658; PMCID: PMC2715965.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCells
    186. Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies. J Pediatr Hematol Oncol. 2008 Jun; 30(6):411-8. Shah AJ, Epport K, Azen C, Killen R, Wilson K, De Clerck D, Crooks G, Kapoor N, Kohn DB, Parkman R, Weinberg KI. PMID: 18525456.
      View in: PubMed   Mentions: 29     Fields:    Translation:Humans
    187. In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol Ther. 2008 Jul; 16(7):1308-15. Bauer G, Dao MA, Case SS, Meyerrose T, Wirthlin L, Zhou P, Wang X, Herrbrich P, Arevalo J, Csik S, Skelton DC, Walker J, Pepper K, Kohn DB, Nolta JA. PMID: 18461052; PMCID: PMC3013368.
      View in: PubMed   Mentions: 38     Fields:    Translation:AnimalsCells
    188. Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system. Cancer Res. 2008 Apr 15; 68(8):2961-71. Singh H, Manuri PR, Olivares S, Dara N, Dawson MJ, Huls H, Hackett PB, Kohn DB, Shpall EJ, Champlin RE, Cooper LJ. PMID: 18413766; PMCID: PMC2424272.
      View in: PubMed   Mentions: 141     Fields:    Translation:HumansCells
    189. Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion. Blood. 2008 Jun 15; 111(12):5745-54. Carbonaro DA, Jin X, Cotoi D, Mi T, Yu XJ, Skelton DC, Dorey F, Kellems RE, Blackburn MR, Kohn DB. PMID: 18356486; PMCID: PMC2424166.
      View in: PubMed   Mentions: 11     Fields:    Translation:AnimalsCells
    190. Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl. Blood. 2008 Apr 15; 111(8):4064-74. Abdel-Azim H, Zhu Y, Hollis R, Wang X, Ge S, Hao QL, Smbatyan G, Kohn DB, Rosol M, Crooks GM. PMID: 18174381; PMCID: PMC2288719.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansAnimalsCells
    191. Human hematopoietic cell culture, transduction, and analyses. Curr Protoc Hum Genet. 2008 Jan; Chapter 13:Unit 13.7. Bonde J, Wirthlin L, Kohn DB, Nolta JA. PMID: 18428422.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    192. Genetic therapies against HIV. Nat Biotechnol. 2007 Dec; 25(12):1444-54. Rossi JJ, June CH, Kohn DB. PMID: 18066041; PMCID: PMC4539027.
      View in: PubMed   Mentions: 110     Fields:    Translation:Humans
    193. Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types. Exp Biol Med (Maywood). 2007 Nov; 232(10):1368-80. Rodriguez RT, Velkey JM, Lutzko C, Seerke R, Kohn DB, O'Shea KS, Firpo MT. PMID: 17959850.
      View in: PubMed   Mentions: 22     Fields:    Translation:HumansCells
    194. Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication. Mol Ther. 2008 Jan; 16(1):46-51. Taylor JA, Vojtech L, Bahner I, Kohn DB, Laer DV, Russell DW, Richard RE. PMID: 17955023.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCells
    195. Preferential association of prostate cancer cells expressing prostate specific membrane antigen to bone marrow matrix. Int J Oncol. 2007 Apr; 30(4):899-904. Barwe SP, Maul RS, Christiansen JJ, Anilkumar G, Cooper CR, Kohn DB, Rajasekaran AK. PMID: 17332929.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansCells
    196. The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants. Biol Blood Marrow Transplant. 2007 May; 13(5):584-93. Shah AJ, Kapoor N, Crooks GM, Weinberg KI, Azim HA, Killen R, Kuo L, Rushing T, Kohn DB, Parkman R. PMID: 17448918.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCellsCTClinical Trials
    197. Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Mol Ther. 2007 Jan; 15(1):76-85. Bahner I, Sumiyoshi T, Kagoda M, Swartout R, Peterson D, Pepper K, Dorey F, Reiser J, Kohn DB. PMID: 17164778.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansCells
    198. Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Mol Ther. 2007 Jan; 15(1):183-92. Blumenthal M, Skelton D, Pepper KA, Jahn T, Methangkool E, Kohn DB. PMID: 17164790.
      View in: PubMed   Mentions: 15     Fields:    Translation:AnimalsCells
    199. Lentiviral vectors ready for prime-time. Nat Biotechnol. 2007 Jan; 25(1):65-6. Kohn DB. PMID: 17211402.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCells
    200. Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Exp Hematol. 2006 Oct; 34(10):1333-43. Hollis RP, Nightingale SJ, Wang X, Pepper KA, Yu XJ, Barsky L, Crooks GM, Kohn DB. PMID: 16982326.
      View in: PubMed   Mentions: 19     Fields:    Translation:HumansCells
    201. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood. 2007 Jan 15; 109(2):503-6. Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, Hershfield MS, Candotti F, Kohn DB. PMID: 16973956; PMCID: PMC1785101.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansCells
    202. Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targeting to tumor vasculature. Microvasc Res. 2006 Jul-Sep; 72(1-2):54-61. Anilkumar G, Barwe SP, Christiansen JJ, Rajasekaran SA, Kohn DB, Rajasekaran AK. PMID: 16713605.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansCells
    203. In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: a novel form of enzyme replacement therapy for ADA deficiency. Mol Ther. 2006 Jun; 13(6):1110-20. Carbonaro DA, Jin X, Petersen D, Wang X, Dorey F, Kil KS, Aldrich M, Blackburn MR, Kellems RE, Kohn DB. PMID: 16651028.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansAnimalsCells
    204. Transient gene expression by nonintegrating lentiviral vectors. Mol Ther. 2006 Jun; 13(6):1121-32. Nightingale SJ, Hollis RP, Pepper KA, Petersen D, Yu XJ, Yang C, Bahner I, Kohn DB. PMID: 16556511.
      View in: PubMed   Mentions: 84     Fields:    Translation:HumansCells
    205. Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector. Exp Hematol. 2006 Mar; 34(3):369-81. Kahl CA, Tarantal AF, Lee CI, Jimenez DF, Choi C, Pepper K, Petersen D, Fletcher MD, Leapley AC, Fisher J, Burns TS, Ultsch MN, Dorey FJ, Kohn DB. PMID: 16543071.
      View in: PubMed   Mentions: 19     Fields:    Translation:HumansAnimalsCells
    206. Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein. J Virol. 2006 Feb; 80(4):1863-73. Unwalla HJ, Li HT, Bahner I, Li MJ, Kohn D, Rossi JJ. PMID: 16439542; PMCID: PMC1367144.
      View in: PubMed   Mentions: 27     Fields:    Translation:HumansCells
    207. Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors. Stem Cells Dev. 2006 Feb; 15(1):109-17. Jang JE, Shaw K, Yu XJ, Petersen D, Pepper K, Lutzko C, Kohn DB. PMID: 16522168.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansAnimalsCells
    208. Perspectives on gene therapy for immune deficiencies. Biol Blood Marrow Transplant. 2005 Dec; 11(12):972-6. Podsakoff GM, Engel BC, Kohn DB. PMID: 16338618.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansAnimals
    209. Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen. Blood. 2006 Mar 15; 107(6):2286-93. Mitsuhashi N, Fischer-Lougheed J, Shulkin I, Kleihauer A, Kohn DB, Weinberg KI, Starnes VA, Kearns-Jonker M. PMID: 16291598.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    210. Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B. Pediatrics. 2005 Oct; 116(4):1022-5. Shah AJ, Kapoor N, Crooks GM, Parkman R, Weinberg KI, Wilson K, Kohn DB. PMID: 16199719.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    211. Recombinant murine interleukin-12 elicits potent antileukemic immune responses in a murine model of Philadelphia chromosome-positive acute lymphoblastic leukemia. Cancer Gene Ther. 2005 Oct; 12(10):818-24. Gruber TA, Skelton DC, Kohn DB. PMID: 15877085.
      View in: PubMed   Mentions: 2     Fields:    Translation:AnimalsCellsPHPublic Health
    212. Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clin Immunol. 2005 Nov; 117(2):133-43. Chan B, Wara D, Bastian J, Hershfield MS, Bohnsack J, Azen CG, Parkman R, Weinberg K, Kohn DB. PMID: 16112907.
      View in: PubMed   Mentions: 57     Fields:    Translation:HumansCells
    213. Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Mol Ther. 2005 Jul; 12(1):77-86. Podsakoff GM, Engel BC, Carbonaro DA, Choi C, Smogorzewska EM, Bauer G, Selander D, Csik S, Wilson K, Betts MR, Koup RA, Nabel GJ, Bishop K, King S, Schmidt M, von Kalle C, Church JA, Kohn DB. PMID: 15963923.
      View in: PubMed   Mentions: 42     Fields:    Translation:HumansCells
    214. Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther. 2005 Jul; 12(1):87-98. Tarantal AF, McDonald RJ, Jimenez DF, Lee CC, O'Shea CE, Leapley AC, Won RH, Plopper CG, Lutzko C, Kohn DB. PMID: 15963924.
      View in: PubMed   Mentions: 31     Fields:    Translation:AnimalsCells
    215. Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Mol Ther. 2005 May; 11(5):776-89. Kobayashi H, Carbonaro D, Pepper K, Petersen D, Ge S, Jackson H, Shimada H, Moats R, Kohn DB. PMID: 15851016.
      View in: PubMed   Mentions: 50     Fields:    Translation:HumansAnimalsCells
    216. Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Hum Gene Ther. 2005 Apr; 16(4):417-25. Lee CC, Jimenez DF, Kohn DB, Tarantal AF. PMID: 15871673.
      View in: PubMed   Mentions: 17     Fields:    Translation:AnimalsCells
    217. Factors influencing the titer and infectivity of lentiviral vectors. Hum Gene Ther. 2004 Oct; 15(10):976-88. Logan AC, Nightingale SJ, Haas DL, Cho GJ, Pepper KA, Kohn DB. PMID: 15585113.
      View in: PubMed   Mentions: 38     Fields:    Translation:HumansCells
    218. Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration. J Virol. 2004 Aug; 78(16):8421-36. Logan AC, Haas DL, Kafri T, Kohn DB. PMID: 15280451; PMCID: PMC479072.
      View in: PubMed   Mentions: 27     Fields:    Translation:HumansCells
    219. Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Mol Genet Metab. 2004 Aug; 82(4):286-95. Zheng Y, Ryazantsev S, Ohmi K, Zhao HZ, Rozengurt N, Kohn DB, Neufeld EF. PMID: 15308126.
      View in: PubMed   Mentions: 24     Fields:    Translation:AnimalsCells
    220. Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts. Transplantation. 2004 Jun 15; 77(11):1748-54. Kearns-Jonker M, Fischer-Lougheed J, Shulkin I, Kleihauer A, Mitsuhashi N, Kohn DB, Weinberg K, D'Apice AJ, Starnes VA, Cramer DV. PMID: 15201677.
      View in: PubMed   Mentions: 6     Fields:    Translation:AnimalsCells
    221. Simian immunodeficiency virus infection of hematopoietic stem cells and bone marrow stromal cells. J Acquir Immune Defic Syndr. 2004 May 01; 36(1):553-61. Lee CI, Cowan MJ, Kohn DB, Tarantal AF. PMID: 15097297.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    222. Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation. J Pediatr Hematol Oncol. 2004 Feb; 26(2):91-7. Shah AJ, Lenarsky C, Kapoor N, Crooks GM, Kohn DB, Parkman R, Epport K, Wilson K, Weinberg K. PMID: 14767194.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    223. Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells. Mol Ther. 2004 Jan; 9(1):112-23. Lee CI, Kohn DB, Ekert JE, Tarantal AF. PMID: 14741784.
      View in: PubMed   Mentions: 15     Fields:    Translation:AnimalsCells
    224. Update on gene therapy of inherited immune deficiencies. Curr Opin Mol Ther. 2003 Oct; 5(5):503-7. Engel BC, Kohn DB, Podsakoff GM. PMID: 14601519.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansCells
    225. The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells. J Virol. 2003 Sep; 77(17):9439-50. Haas DL, Lutzko C, Logan AC, Cho GJ, Skelton D, Jin Yu X, Pepper KA, Kohn DB. PMID: 12915559; PMCID: PMC187403.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansAnimalsCells
    226. Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines. Mol Cancer Ther. 2003 Sep; 2(9):911-7. Goto H, Yang B, Petersen D, Pepper KA, Alfaro PA, Kohn DB, Reynolds CP. PMID: 14555710.
      View in: PubMed   Mentions: 21     Fields:    Translation:HumansCells
    227. Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging. Blood. 2003 Nov 15; 102(10):3478-82. Wang X, Rosol M, Ge S, Peterson D, McNamara G, Pollack H, Kohn DB, Nelson MD, Crooks GM. PMID: 12946998.
      View in: PubMed   Mentions: 57     Fields:    Translation:HumansAnimalsCells
    228. Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Mol Genet Metab. 2003 Aug; 79(4):233-44. Zheng Y, Rozengurt N, Ryazantsev S, Kohn DB, Satake N, Neufeld EF. PMID: 12948739.
      View in: PubMed   Mentions: 40     Fields:    Translation:AnimalsCells
    229. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther. 2003 Aug; 8(2):180-7. Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem HP, Candotti F, Tisdale J, Riviére I, Blau CA, Richard RE, Sorrentino B, Nolta J, Malech H, Brenner M, Cornetta K, Cavagnaro J, High K, Glorioso J, American Society of Gene Therapy (ASGT). PMID: 12907140.
      View in: PubMed   Mentions: 30     Fields:    Translation:HumansAnimalsCells
    230. Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells. Blood. 2003 Nov 01; 102(9):3117-9. Kurre P, Morris J, Thomasson B, Kohn DB, Kiem HP. PMID: 12869503.
      View in: PubMed   Mentions: 10     Fields:    Translation:AnimalsCells
    231. Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes. J Virol. 2003 Jul; 77(13):7341-51. Lutzko C, Senadheera D, Skelton D, Petersen D, Kohn DB. PMID: 12805432; PMCID: PMC164820.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansAnimalsCells
    232. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer. 2003 Jul; 3(7):477-88. Kohn DB, Sadelain M, Glorioso JC. PMID: 12835668.
      View in: PubMed   Mentions: 98     Fields:    Translation:Humans
    233. Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT). J Gene Med. 2003 Jul; 5(7):641. Kohn DB, Gänsbacher B. PMID: 12825204.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    234. Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Ann N Y Acad Sci. 2003 May; 996:112-21. Schmidt M, Glimm H, Wissler M, Hoffmann G, Olsson K, Sellers S, Carbonaro D, Tisdale JF, Leurs C, Hanenberg H, Dunbar CE, Kiem HP, Karlsson S, Kohn DB, Williams D, Von Kalle C. PMID: 12799289.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansAnimalsCells
    235. Effects of the negative control region on expression from retroviral LTR. Mol Ther. 2003 Apr; 7(4):438-40. Wang L, Haas D, Halene S, Kohn DB. PMID: 12727105.
      View in: PubMed   Mentions: 2     Fields:    Translation:AnimalsCells
    236. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat Med. 2003 Apr; 9(4):463-8. Schmidt M, Carbonaro DA, Speckmann C, Wissler M, Bohnsack J, Elder M, Aronow BJ, Nolta JA, Kohn DB, von Kalle C. PMID: 12640448.
      View in: PubMed   Mentions: 45     Fields:    Translation:HumansCells
    237. [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]. Med Wieku Rozwoj. 2003 Jan-Mar; 7(1):27-34. Smogorzewska EM, Weinberg KI, Kohn DB. PMID: 13130167.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    238. Gene therapy for inborn and acquired immune deficiency disorders. Acta Haematol. 2003; 110(2-3):60-70. Engel BC, Kohn DB. PMID: 14583666.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    239. Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr Opin Biotechnol. 2002 Oct; 13(5):429-36. Logan AC, Lutzko C, Kohn DB. PMID: 12459333.
      View in: PubMed   Mentions: 26     Fields:    Translation:HumansAnimalsCells
    240. Kinetics of fluorescence expression in nonhuman primates transplanted with GFP retrovirus-modified CD34 cells. Mol Ther. 2002 Jul; 6(1):83-90. Kurre P, Morris J, Andrews RG, Kohn DB, Kiem HP. PMID: 12095307.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    241. Requirement for NK cells in CD40 ligand-mediated rejection of Philadelphia chromosome-positive acute lymphoblastic leukemia cells. J Immunol. 2002 Jan 01; 168(1):73-80. Gruber TA, Skelton DC, Kohn DB. PMID: 11751948.
      View in: PubMed   Mentions: 3     Fields:    Translation:AnimalsCells
    242. Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up. Biol Blood Marrow Transplant. 2002; 8(4):221-8. Shah AJ, Kapoor N, Weinberg KI, Crooks GM, Kohn DB, Lenarsky C, Kaufman F, Epport K, Wilson K, Parkman R. PMID: 12014811.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    With one click, see people who:

    Run Clinical Trials

    See more options in the Advanced Search tab

    Donald's Networks
    Concepts (724)
    Derived automatically from this person's publications.
    Explore
    _
    Co-Authors (110)
    People in Profiles who have published with this person.
    Explore
    _
    Similar People (60)
    People who share similar concepts with this person.
    Explore
    _
    Same Department
    Search Department
    _

    UCLA Profiles is managed by the UCSF Clinical and Translational Science Institute (CTSI), part of the Clinical and Translational Science Award program funded by the National Center for Advancing Translational Sciences (Grant Number UL1 TR000004) at the National Institutes of Health (NIH).
    This site is running Profiles RNS version UCSF-v3.1.0-3-g1ef1264b on PROFILES-PWEB03. We use cookies to operate our website. We also use cookies to analyze our site’s performance and improve your experience on our site. For more information about how we use cookies, please see our Website Terms of Use.

    Copyright © The Regents of the University of California