Dr. Melissa Spencer is a Professor of Neurology at UCLA. Dr. Spencers 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.
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.
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.
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
Journal of lipid research.
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.
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.
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.
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. .
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.
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
Cell stem cell.