Melissa J. Spencer, Ph.D. [ Edit Your Profile ]

Email Address:

Fax Number: 310-206-1998
Lab Number: (310) 267-4582
Office Phone Number: 310-794-5225

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

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



Dr. Melissa Spencer is a Professor of Neurology at UCLA. Dr. Spencer’s research expertise is in the area of pathogenesis of muscular dystrophy using genetically modified mouse models. She has had a career long interest in pathogenesis of muscular dystrophy (DMD and LGMD) and has extensive experience in generating and phenotyping genetically modified mouse models (transgenic, knock out and knock in models). A particular strength of the Spencer lab is the longitudinal and multidisciplinary approach used to study these muscular dystrophies that ranges from the generation of genetically modified mice to use of biochemical, cell biological, immunological and functional approaches for analyzing phenotypic outcomes. One of these studies was carried into two clinical trials at UCLA, in which Dr. Spencer was the principal investigator, with Dr. Fowler who was a co-investigator; thus, the researchers at the UCLA MDCRC are truly performing "bench-to-bedside translational research". Dr. Spencer was the recipient of a PECASE (Presidential Early Career Award For Scientists and Engineers) in 2001. 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. Dr. Spencer brings a strong translational research perspective to the Center for the application of HTS and Genomic approaches to muscular dystrophy research.

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): .
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.
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 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.
Mokhonova Ekaterina I, Avliyakulov Nuraly K, Kramerova Irina, Kudryashova Elena, Haykinson Michael J, Spencer Melissa J, The E3 ubiquitin ligase TRIM32 regulates myoblast proliferation by controlling turnover of NDRG2. Human molecular genetics. 2015; 24(10): 2873-83.
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.
Chao Lily C, Wroblewski Kevin, Ilkayeva Olga R, Stevens Robert D, Bain James, Meyer Gretchen A, Schenk Simon, Martinez Leonel, Vergnes Laurent, Narkar Vihang A, Drew Brian G, Hong Cynthia, Boyadjian Rima, Hevener Andrea L, Evans Ronald M, Reue Karen, Spencer Melissa J, Newgard Christopher B, Tontonoz Peter, Skeletal muscle Nur77 expression enhances oxidative metabolism and substrate utilization. Journal of lipid research. 2012; 53(12): 2610-9.
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.
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.
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.
Shieh Perry B, Kudryashova Elena, Spencer Melissa J, Limb-girdle muscular dystrophy 2H and the role of TRIM32. Handbook of clinical neurology / edited by P.J. Vinken and G.W. Bruyn. 2011; 101(7): 125-33.
Kim Michelle H, Kay Danielle I, Rudra Renuka T, Chen Bo Ming, Hsu Nigel, Izumiya Yasuhiro, Martinez Leonel, Spencer Melissa J, Walsh Kenneth, Grinnell Alan D, Crosbie Rachelle H, Myogenic Akt signaling attenuates muscular degeneration, promotes myofiber regeneration and improves muscle function in dystrophin-deficient mdx mice. Human molecular genetics. 2011; 20(7): 1324-38.
Mellgren Ronald L, Miyake Katsuya, Kramerova Irina, Spencer Melissa J, Bourg Nathalie, Bartoli Marc, Richard Isabelle, Greer Peter A, McNeil Paul L, Calcium-dependent plasma membrane repair requires m- or mu-calpain, but not calpain-3, the proteasome, or caspases. Biochimica et biophysica acta. 2009; 1793(12): 1886-93.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Guyon, JR Kudryashova, E Potts, A Dalkilic, I Brosius, MA Thompson, TG Beckmann, JS Kunkel, LM Spencer, MJ, Calpain 3 cleaves filamin C and regulates its ability to interact with gamma- and delta-sarcoglycans. Muscle & nerve. . 2003; 28(4): 472-83.
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, Skipping to new gene therapies for muscular dystrophy. Nature Medicine. 2003; 9(8): 997-8.
Tidball James G, Spencer Melissa J, Skipping to new gene therapies for muscular dystrophy. Nature medicine. 2003; 9(8): 997-8.
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.
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.
Spencer, MJ Mellgren, RL, Overexpression of a calpastatin transgene in mdx muscle reduces dystrophic pathology. Human molecular genetics. . 2002; 11(21): 2645-55.
Spencer, M.J., Guyon, J.R., Sorimachi, H., Potts, A., Richard, I., Herasse, M., Chamberlain, J., Dalkilic, I., Kunkel, L.M., and Beckmann, J.S., Stable expression of calpain 3 from a muscle transgene in vivo; immature muscle in transgenic mice suggests a role for calpain 3 in muscle maturation. Proceedings of the National Academy of Sciences. 2002; 99(3): 8874-8879.
Taveau Mathieu, Stockholm Daniel, Spencer Melissa, Richard Isabelle, Quantification of splice variants using molecular beacon or scorpion primers. Analytical biochemistry. 2002; 305(2): 227-35.
Spencer, MJ Montecino-Rodriguez, E Dorshkind, K Tidball, JG, Helper (CD4(+)) and cytotoxic (CD8(+)) T cells promote the pathology of dystrophin-deficient muscle. Clinical immunology (Orlando, Fla.) . 2001; 98(2): 235-43.
Spencer, MJ Marino, MW Winckler, WM, Altered pathological progression of diaphragm and quadriceps muscle in TNF-deficient, dystrophin-deficient mice. Neuromuscular disorders : NMD. . 2000; 10(8): 612-9.
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; .