Melissa Spencer, Ph.D.

A Short Biography:

Dr. Melissa Spencer is a Professor of Neurology at UCLA. She performed both her PhD and post-doctoral studies at UCLA, elucidating pathogenic mechanisms of Duchenne Muscular Dystrophy. As faculty in the Department of Neurology, Dr. Spencer’s research program continues to focus on pathogenesis and therapies for Duchenne and Limb Girdle Muscular Dystrophies, using a longitudinal and multidisciplinary approach that ranges from the generation of genetically modified mice to use of biochemical, cell biological, immunological and functional approaches for analyzing phenotypic outcomes. She has lead two clinical trials at UCLA, with Dr. Fowler as co-investigator. The trials assessed the efficacy of Albuterol in Duchenne and provided much of the framework and clinical trial structure that currently exists at UCLA. This foundation has enabled UCLA’s participation in industry and investigator initiated trials for Duchenne. Dr. Spencer was the recipient of a PECASE (Presidential Early Career Award For Scientists and Engineers). She has participated in numerous NIH advisory committees including as a regular standing member of the SMEP study section and she is currently on the Scientific Advisory Committee for the Muscular Dystrophy Association, PPMD and CCC. Dr. Spencer brings a strong translational research perspective to the Center for the application of HTS and Genomic approaches to muscular dystrophy research.

Work Titles
UCLA Professor, Neurology Co-Director, Center for Duchenne Muscular Dystrophy Member, Cell & Developmental Biology GPB Home Area Member, JCCC Signal Transduction and Therapeutics Program Area Member, Molecular, Cellular & Integrative Physiology GPB Home Area

Contact Information:

Fax Number:


Lab Number:

(310) 267-4582

Office Phone Number:


Office Address:

635 Charles Young Dr. So
mail code 703422
Los Angeles, CA 90095

Work Address:

635 Charles E. Young Dr. So
mail code 733422
Los Angeles, CA 90095

Detailed Biography:

The focus of the Spencer lab is pathogenesis of muscular dystrophy using genetically modified mouse models. Genetically modified mice (transgenic, knock out and knock in models) are used to better understand the muscular dystrophies, develop therapies and to learn about normal muscle function. A variety of approaches are used to study these diseases that range from use of biochemical, cell biological, immunological and functional approaches for analyzing phenotypic outcomes. Currently, there are three ongoing projects in the Spencer lab. First, the lab is focused on understanding the signaling pathways that are impacted in calpainopathy (limb girdle muscular dystrophy type 2A) including CaMK and TK signaling. The lab also is involved in evaluating the contribution of immune cells to the development of fibrosis in Duchenne muscular dystrophy (DMD). Finally,in collaboration with Drs. Carrie Miceli and Stanley Nelson, the lab is involved in identifying strategies to improve efficiency of exon skipping in DMD.


A selected list of publications:

Young Courtney S, Hicks Michael R, Ermolova Natalia V, Nakano Haruko, Jan Majib, Younesi Shahab, Karumbayaram Saravanan, Kumagai-Cresse Chino, Wang Derek, Zack Jerome A, Kohn Donald B, Nakano Atsushi, Nelson Stanley F, Miceli M Carrie, Spencer Melissa J, Pyle April D   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; .
Villalta SA, Rosenthal W, Martinez L, Kaur A, Sparwasser T, Tidball JG, Margeta M, Spencer MJ, Bluestone JA.   Regulatory T cells suppress muscle inflammation and injury in muscular dystrophy, Science Translational Medicine, ; 6(258): .
Martinez Leonel, Ermolova Natalia V, Ishikawa Tomo-O, Stout David B, Herschman Harvey R, Spencer Melissa J   A reporter mouse for optical imaging of inflammation in mdx muscles Skeletal muscle, 2015; 5(2): 15.
Ermolova NV, Martinez L, Vetrone SA, Jordan MC, Roos KP, Sweeney HL, Spencer MJ.   Long-term administration of the TNF blocking drug Remicade (cV1q) to mdx mice reduces skeletal and cardiac muscle fibrosis, but negatively impacts cardiac function, Neuromuscular Disorders, 2014; 24(7): 583-595.
Ermolova Natalia, Kramerova Irina, Spencer Melissa J   Autolytic activation of calpain 3 proteinase is facilitated by calmodulin protein The Journal of biological chemistry, 2015; 290(2): 996-1004.
Kendall Genevieve C, Mokhonova Ekaterina I, Moran Miriana, Sejbuk Natalia E, Wang Derek W, Silva Oscar, Wang Richard T, Martinez Leonel, Lu Qi L, Damoiseaux Robert, Spencer Melissa J, Nelson Stanley F, Miceli M Carrie   Dantrolene enhances antisense-mediated exon skipping in human and mouse models of duchenne muscular dystrophy Science translational medicine, 2012; 4(164): 164ra160.
Kramerova I, Kudryashova E, Ermolova N, Saenz A, Jaka O, Lopez de Munain A, Spencer M J   Impaired calcium calmodulin kinase signaling and muscle adaptation response in the absence of calpain 3 Human molecular genetics, 2012; 21(14): 3193-204.
Kudryashova Elena, Kramerova Irina, Spencer Melissa J   Satellite cell senescence underlies myopathy in a mouse model of limb-girdle muscular dystrophy 2H The Journal of clinical investigation, 2012; 122(5): 1764-76.
Ermolova Natalia, Kudryashova Elena, DiFranco Marino, Vergara Julio, Kramerova Irina, Spencer Melissa J   Pathogenity of some limb girdle muscular dystrophy mutations can result from reduced anchorage to myofibrils and altered stability of calpain 3 Human molecular genetics, 2011; 20(17): 3331-45.
Kudryashova Elena, Struyk Arie, Mokhonova Ekaterina, Cannon Stephen C, Spencer Melissa J   The common missense mutation D489N in TRIM32 causing limb girdle muscular dystrophy 2H leads to loss of the mutated protein in knock-in mice resulting in a Trim32-null phenotype Human molecular genetics, 2011; 20(20): 3925-32.
Kudryashova Elena, Wu Jun, Havton Leif A, Spencer Melissa J   Deficiency of the E3 ubiquitin ligase TRIM32 in mice leads to a myopathy with a neurogenic component Human molecular genetics, 2009; 18(7): 1353-67.
Nelson Stanley F, Crosbie Rachelle H, Miceli M Carrie, Spencer Melissa J   Emerging genetic therapies to treat Duchenne muscular dystrophy Current opinion in neurology, 2009; 22(5): 532-8.
Kramerova Irina, Kudryashova Elena, Wu Benjamin, Germain Sean, Vandenborne Krista, Romain Nadine, Haller Ronald G, Verity M Anthony, Spencer Melissa J   Mitochondrial abnormalities, energy deficit and oxidative stress are features of calpain 3 deficiency in skeletal muscle Human molecular genetics, 2009; 18(17): 3194-205.
Vetrone Sylvia A, Montecino-Rodriguez Encarnacion, Kudryashova Elena, Kramerova Irina, Hoffman Eric P, Liu Scot D, Miceli M Carrie, Spencer Melissa J   Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta The Journal of clinical investigation, 2009; 119(6): 1583-94.
Skura, C. L. Fowler, E. G. Wetzel, G. T. Graves, M. Spencer, M. J.   Albuterol increases lean body mass in ambulatory boys with Duchenne or Becker muscular dystrophy Neurology, 2008; 70(2): 137-43.
Beckmann Jacques S, Spencer Melissa   Calpain 3, the "gatekeeper" of proper sarcomere assembly, turnover and maintenance Neuromuscular disorders : NMD, 2008; 18(12): 913-21.
Kramerova Irina, Kudryashova Elena, Wu Benjamin, Ottenheijm Coen, Granzier Henk, Spencer Melissa J   Novel role of calpain-3 in the triad-associated protein complex regulating calcium release in skeletal muscle Human molecular genetics, 2008; 17(21): 3271-80.
Groshong Jason S, Spencer Melissa J, Bhattacharyya Bula J, Kudryashova Elena, Vohra Bhupinder P S, Zayas Roberto, Wollmann Robert L, Miller Richard J, Gomez Christopher M   Calpain activation impairs neuromuscular transmission in a mouse model of the slow-channel myasthenic syndrome The Journal of clinical investigation, 2007; 117(10): 2903-12.
Kramerova Irina, Beckmann Jacques S, Spencer Melissa J   Molecular and cellular basis of calpainopathy (limb girdle muscular dystrophy type 2A) Biochimica et biophysica acta, 2007; 1772(2): 128-44.
Cohen Niaz, Kudryashova Elena, Kramerova Irina, Anderson Louise V B, Beckmann Jacques S, Bushby Katherine, Spencer Melissa J   Identification of putative in vivo substrates of calpain 3 by comparative proteomics of overexpressing transgenic and nontransgenic mice Proteomics, 2006; 6(22): 6075-84.
Kramerova Irina, Kudryashova Elena, Wu Benjamin, Spencer Melissa J   Regulation of the M-cadherin-beta-catenin complex by calpain 3 during terminal stages of myogenic differentiation Molecular and cellular biology, 2006; 26(22): 8437-47.
Nazarian Ramin, Starcevic Marta, Spencer Melissa J, Dell'Angelica Esteban C   Reinvestigation of the dysbindin subunit of BLOC-1 (biogenesis of lysosome-related organelles complex-1) as a dystrobrevin-binding protein The Biochemical journal, 2006; 395(3): 587-98.
Kramerova Irina, Kudryashova Elena, Venkatraman Gayathri, Spencer Melissa J   Calpain 3 participates in sarcomere remodeling by acting upstream of the ubiquitin-proteasome pathway Human molecular genetics, 2005; 14(15): 2125-34.
Huebsch Kimberly A, Kudryashova Elena, Wooley Christine M, Sher Roger B, Seburn Kevin L, Spencer Melissa J, Cox Gregory A   Mdm muscular dystrophy: interactions with calpain 3 and a novel functional role for titin's N2A domain Human molecular genetics, 2005; 14(19): 2801-11.
Kudryashova Elena, Kudryashov Dmitri, Kramerova Irina, Spencer Melissa J   Trim32 is a ubiquitin ligase mutated in limb girdle muscular dystrophy type 2H that binds to skeletal muscle myosin and ubiquitinates actin Journal of molecular biology, 2005; 354(2): 413-24.
Kramerova I, Kudryashova E, Tidball J G, Spencer Melissa J   Null mutation of calpain 3 (p94) in mice causes abnormal sarcomere formation in vivo and in vitro Human molecular genetics, 2004; 13(13): 1373-88.
Fowler, EG Graves, MC Wetzel, GT Spencer, MJ   Pilot trial of albuterol in Duchenne and Becker muscular dystrophy Neurology. , 2004; 62(6): 1006-8.
Hodgetts Stuart I, Spencer Melissa J, Grounds Miranda D   A role for natural killer cells in the rapid death of cultured donor myoblasts after transplantation Transplantation, 2003; 75(6): 863-71.
Guyon Jeffrey R, Kudryashova Elena, Potts Alexandra, Dalkilic Isin, Brosius Melissa A, Thompson Terri G, Beckmann Jacques S, Kunkel Louis M, Spencer Melissa J   Calpain 3 cleaves filamin C and regulates its ability to interact with gamma- and delta-sarcoglycans Muscle & nerve, 2003; 28(4): 472-83.
Tidball James G, Spencer Melissa J   Skipping to new gene therapies for muscular dystrophy Nature medicine, 2003; 9(8): 997-8.
Gosselin Luc E, Barkley Jacob E, Spencer Melissa J, McCormick Kathleen M, Farkas Gaspar A   Ventilatory dysfunction in mdx mice: impact of tumor necrosis factor-alpha deletion Muscle & nerve, 2003; 28(3): 336-43.
Tidball James G, Spencer Melissa J   Expression of a calpastatin transgene slows muscle wasting and obviates changes in myosin isoform expression during murine muscle disuse The Journal of physiology, 2002; 545(Pt 3): 819-28.
Spencer Melissa J, Mellgren Ronald L   Overexpression of a calpastatin transgene in mdx muscle reduces dystrophic pathology Human molecular genetics, 2002; 11(21): 2645-55.

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