AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

Objective Dysferlinopathies are a family of untreatable muscle disorders caused by mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy with chronic muscle fiber loss, inflammation, fat replacement, and fibrosis; leading to deteriorating muscle weakness. The ob...

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Veröffentlicht in:	Annals of clinical and translational neurology 2015-03, Vol.2 (3), p.256-270
Hauptverfasser:	Sondergaard, Patricia C., Griffin, Danielle A., Pozsgai, Eric R., Johnson, Ryan W., Grose, William E., Heller, Kristin N., Shontz, Kim M., Montgomery, Chrystal L., Liu, Joseph, Clark, Kelly Reed, Sahenk, Zarife, Mendell, Jerry R., Rodino‐Klapac, Louise R.
Format:	Artikel
Sprache:	eng
Schlagworte:	Amino acids Enzymes Experiments Gene expression Gene therapy Muscular dystrophy Musculoskeletal system Mutation Proteins Toxicology Vectors (Biology)
Online-Zugang:	Volltext
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