Abstract

The investigator proposes to study the function and assembly of the dystrophin and utrophin associated protein (DAP/UAP) complexes in skeletal muscle. Regions of dystrophin required for formation of stable DAP/UAP complexes will be identified, DAP/UAP complexes will be identified, DAP/UAP complex associate cytoskeletal or membrane proteins will be isolated, a novel associated protein will be studied in detail. The investigator will employ transgenic and knock-out mouse technologies to analyze function of key protein, as well as immunological and biochemical fractionation together with yeast two-hybrid studies to identify and characterize interactions between associated proteins.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Research Project (R01)
Project #
5R01AR044533-03
Application #
2899917
Study Section
Biological Sciences 2 (BIOL)
Program Officer
Lymn, Richard W
Project Start
1997-04-19
Project End
2002-03-31
Budget Start
1999-04-01
Budget End
2000-03-31
Support Year
3
Fiscal Year
1999
Total Cost
Indirect Cost

  Institution

Name
University of Michigan Ann Arbor
Department
Genetics
Type
Schools of Medicine
DUNS #
791277940
City
Ann Arbor
State
MI
Country
United States
Zip Code
48109

  Publications

Crudele, Julie M; Chamberlain, Jeffrey S (2018) Cas9 immunity creates challenges for CRISPR gene editing therapies. Nat Commun 9:3497
Bengtsson, Niclas E; Hall, John K; Odom, Guy L et al. (2017) Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Nat Commun 8:14454
Bengtsson, Niclas E; Hall, John K; Odom, Guy L et al. (2017) Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Nat Commun 8:16007
Bengtsson, Niclas E; Seto, Jane T; Hall, John K et al. (2016) Progress and prospects of gene therapy clinical trials for the muscular dystrophies. Hum Mol Genet 25:R9-17
Davey, Jonathan R; Watt, Kevin I; Parker, Benjamin L et al. (2016) Integrated expression analysis of muscle hypertrophy identifies Asb2 as a negative regulator of muscle mass. JCI Insight 1:
Su, Wei; Kang, John; Sopher, Bryce et al. (2016) Recombinant adeno-associated viral (rAAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia. J Neurochem 136 Suppl 1:49-62
Muir, Lindsey A; Murry, Charles E; Chamberlain, Jeffrey S (2016) Prosurvival Factors Improve Functional Engraftment of Myogenically Converted Dermal Cells into Dystrophic Skeletal Muscle. Stem Cells Dev :
Hollinger, Katrin; Chamberlain, Jeffrey S (2015) Viral vector-mediated gene therapies. Curr Opin Neurol 28:522-7
Ramos, Julian; Chamberlain, Jeffrey S (2015) Gene Therapy for Duchenne muscular dystrophy. Expert Opin Orphan Drugs 3:1255-1266
Banks, Glen B; Combs, Ariana C; Odom, Guy L et al. (2014) Muscle structure influences utrophin expression in mdx mice. PLoS Genet 10:e1004431

Showing the most recent 10 out of 34 publications