Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
1U54AR065139-01A1
Application #
8736372
Study Section
Special Emphasis Panel (ZAR1-KM (M1))
Project Start
Project End
Budget Start
2014-04-01
Budget End
2015-03-31
Support Year
1
Fiscal Year
2014
Total Cost
$172,196
Indirect Cost
$56,759
Name
University of Washington
Department
Type
DUNS #
605799469
City
Seattle
State
WA
Country
United States
Zip Code
98195
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
Mack, David L; Poulard, Karine; Goddard, Melissa A et al. (2017) Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs. Mol Ther 25:839-854
Chamberlain, Joel R; Chamberlain, Jeffrey S (2017) Progress toward Gene Therapy for Duchenne Muscular Dystrophy. Mol Ther 25:1125-1131
Amoasii, Leonela; Long, Chengzu; Li, Hui et al. (2017) Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy. Sci Transl Med 9:
Elverman, Matthew; Goddard, Melissa A; Mack, David et al. (2017) Long-term effects of systemic gene therapy in a canine model of myotubular myopathy. Muscle Nerve 56:943-953
Whitehead, Nicholas P; Bible, Kenneth L; Kim, Min Jeong et al. (2016) Validation of ultrasonography for non-invasive assessment of diaphragm function in muscular dystrophy. J Physiol 594:7215-7227
Kolwicz Jr, Stephen C; Odom, Guy L; Nowakowski, Sarah G et al. (2016) AAV6-mediated Cardiac-specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility. Mol Ther 24:240-250
Lawlor, Michael W; Beggs, Alan H; Buj-Bello, Ana et al. (2016) Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons. J Neuropathol Exp Neurol 75:102-10
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:

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