The Center for Duchenne Muscular Dystrophy (""""""""CDMD"""""""") provides an academic home in Los Angeles for the conduct of translational research for the muscular dystrophies. Although CDMD supports all muscle and muscular dystrophy related research, its primary focus is Duchenne Muscular Dystrophy (DMD), the most common lethal genetic disorder in children. CDMD unites recognized leaders in basic and clinical muscular dystrophy research with the immense technological and educational infrastructure of UCLA to facilitate discovery and development of new therapeutics for degenerative muscle disease. Our location in Los Angeles, the most populous county in the United States, gives us access to a huge catchment from which to recruit patients with rare muscle diseases studied at CDMD. These resources, coupled with the successes of our existing Center, position CDMD to become the leading translational muscular dystrophy center on the West Coast, if not the nation. Since the first funding of the CDMD Core Center in 2009, the Center has successfully expanded the number of multidisciplinary collaborations focused on muscular dystrophy research and accelerated the discovery and testing of potential therapeutics. In the past four years, we have awarded $650,000 in pilot and feasibility seed grants to 17 labs, and 12 of which went to investigators new to muscle or muscular dystrophy research. Membership in CDMD has doubled to nearly 45 members. Researchers affiliated with our Center have initiated 13 clinical trials and preclinical tests of four therapeutic targets. In this application, we propose to furthe expand our infrastructure and services to create an ecosystem that supports translation of discoveries in muscle and muscular dystrophy research. Through an Administrative Core, an Enrichment Core and 3 basic science cores, CDMD will provide the institutional resources necessary for all aspects of basic science discovery, pre-clinical testing, and clinical trials. Th basic science cores, which include a High Throughput Screening and Cell Repository Core (Core B), a Muscle Phenotyping and Imaging Core (Core C), and a Bioinformatics and Genomics Core (Core D) are designed to facilitate discovery of new therapeutic targets, novel compounds and create models to test these compounds and targets related to muscle disease.

Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Center Core Grants (P30)
Project #
2P30AR057230-06
Application #
8691581
Study Section
Special Emphasis Panel (ZAR1-XZ (M1))
Program Officer
Nuckolls, Glen H
Project Start
2009-04-01
Project End
2019-03-31
Budget Start
2014-04-01
Budget End
2015-03-31
Support Year
6
Fiscal Year
2014
Total Cost
$616,000
Indirect Cost
$216,000
Name
University of California Los Angeles
Department
Neurology
Type
Schools of Medicine
DUNS #
092530369
City
Los Angeles
State
CA
Country
United States
Zip Code
90095
Hicks, Michael R; Hiserodt, Julia; Paras, Katrina et al. (2018) ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs. Nat Cell Biol 20:46-57
Armstrong, Tess; Dregely, Isabel; Stemmer, Alto et al. (2018) Free-breathing liver fat quantification using a multiecho 3D stack-of-radial technique. Magn Reson Med 79:370-382
Wang, Richard T; Barthelemy, Florian; Martin, Ann S et al. (2018) DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype. Hum Mutat 39:1193-1202
Wang, Derek W; Mokhonova, Ekaterina I; Kendall, Genevieve C et al. (2018) Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse. Mol Ther Nucleic Acids 11:180-191
Kramerova, Irina; Torres, Jorge A; Eskin, Ascia et al. (2018) Calpain 3 and CaMKII? signaling are required to induce HSP70 necessary for adaptive muscle growth after atrophy. Hum Mol Genet 27:1642-1653
Aliotta, Eric; Moulin, Kévin; Magrath, Patrick et al. (2018) Quantifying precision in cardiac diffusion tensor imaging with second-order motion-compensated convex optimized diffusion encoding. Magn Reson Med 80:1074-1087
Xi, Haibin; Fujiwara, Wakana; Gonzalez, Karen et al. (2017) In Vivo Human Somitogenesis Guides Somite Development from hPSCs. Cell Rep 18:1573-1585
McMorran, Brian J; Miceli, M Carrie; Baum, Linda G (2017) Lectin-binding characterizes the healthy human skeletal muscle glycophenotype and identifies disease-specific changes in dystrophic muscle. Glycobiology 27:1134-1143
Nakano, Haruko; Minami, Itsunari; Braas, Daniel et al. (2017) Glucose inhibits cardiac muscle maturation through nucleotide biosynthesis. Elife 6:
Gibbs, Elizabeth M; Crosbie-Watson, Rachelle H (2017) A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy. J Vis Exp :

Showing the most recent 10 out of 71 publications