Carmen Bertoni, Ph.D.

Work Titles
California NanoSystems Institute Member, California NanoSystems Institute
UCLA Associate Professor, Neurology

Contact Information:

Mailing Address:

710 Westwood Plaza
4145 RNRC
Los Angeles, CA 90095


A selected list of publications:

Bertoni Carmen   Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells Frontiers in physiology, 2014; 5: 148.
Nik-Ahd Farnoosh, Bertoni Carmen   Ex vivo gene editing of the dystrophin gene in muscle stem cells using peptide nucleic acid single stranded oligodeoxynucleotides (PNA-SsODNs) induces stable expression of dystrophin in a mouse model for Duchenne muscular dystrophy Stem cells (Dayton, Ohio), 2014; .
Cai Lingzhi, Koppanati Bhanu Munil, Bertoni Carmen, Clemens Paula R   In utero delivery of oligodeoxynucleotides for gene correction Methods in molecular biology (Clifton, N.J.), 2014; 1114: 399-411.
Bertoni Carmen   Methods for the assessment of ssODN-mediated gene correction frequencies in muscle cells Methods in molecular biology (Clifton, N.J.), 2014; 1114: 59-83.
Khanjyan Michael V, Yang Jonathan, Kayali Refik, Caldwell Thomas, Bertoni Carmen   A high-content, high-throughput siRNA screen identifies cyclin D2 as a potent regulator of muscle progenitor cell fusion and a target to enhance muscle regeneration Human molecular genetics, 2013; 22(16): 3283-95.
Bertoni Carmen   Therapy development for neuromuscular diseases: translating hope into promise Future neurology, 2013; 8(4): .
Kayali Refik, Ku Jin-Mo, Khitrov Gregory, Jung Michael E, Prikhodko Olga, Bertoni Carmen   Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy Human molecular genetics, 2012; 21(18): 4007-20.
Du Liutao, Kayali Refik, Bertoni Carmen, Fike Francesca, Hu Hailiang, Iversen Patrick L, Gatti Richard A   Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum Human molecular genetics, 2011; 20(16): 3151-60.
Kayali Refik, Bury Frederic, Ballard McIver, Bertoni Carmen   Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs Human molecular genetics, 2010; 19(16): 3266-81.
Bertoni Carmen, Rustagi Arjun, Rando Thomas A   Enhanced gene repair mediated by methyl-CpG-modified single-stranded oligonucleotides Nucleic acids research, 2009; 37(22): 7468-82.
Du Liutao, Damoiseaux Robert, Nahas Shareef, Gao Kun, Hu Hailiang, Pollard Julianne M, Goldstine Jimena, Jung Michael E, Henning Susanne M, Bertoni Carmen, Gatti Richard A   Nonaminoglycoside compounds induce readthrough of nonsense mutations The Journal of experimental medicine, 2009; 206(10): 2285-97.
Bertoni Carmen   Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides Frontiers in bioscience : a journal and virtual library, 2008; 13(10): 517-27.
Portlock Joylette L, Keravala Annahita, Bertoni Carmen, Lee Solomon, Rando Thomas A, Calos Michele P   Long-term increase in mVEGF164 in mouse hindlimb muscle mediated by phage phiC31 integrase after nonviral DNA delivery Human gene therapy, 2006; 17(8): 871-6.
Bertoni Carmen, Jarrahian Sohail, Wheeler Thurman M, Li Yining, Olivares Eric C, Calos Michele P, Rando Thomas A   Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration Proceedings of the National Academy of Sciences of the United States of America, 2006; 103(2): 419-24.
Bertoni Carmen, Morris Glenn E, Rando Thomas A   Strand bias in oligonucleotide-mediated dystrophin gene editing Human molecular genetics, 2005; 14(2): 221-33.
Bertoni Carmen, Lau Catherine, Rando Thomas A   Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping Human molecular genetics, 2003; 12(10): 1087-99.
Bertoni Carmen, Rando Thomas A   Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides Human gene therapy, 2002; 13(6): 707-18.

Does this profile need updating? Contact Us