Adeno-associated viral-mediated LARGE gene therapy rescues the muscular dystrophic phenotype in mouse models of dystroglycanopathy

Human Gene Therapy

Volumn 24, Issue 3, 2013, Pages 317-330

  Yu, Miao ^a   He, Yonglin ^a,b   Wang, Kejian ^a,b   Zhang, Peng ^a   Zhang, Shengle ^a   Hu, Huaiyu ^a  

^a State University of New York Upstate Medical University: College of Medicine   (United States)

^b CHONGQING MEDICAL UNIVERSITY   (China)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA DYSTROGLYCAN; FUKUTIN; GLYCOSYLTRANSFERASE; POMGNT1 PROTEIN; POMT1 PROTEIN; UNCLASSIFIED DRUG; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BINDING AFFINITY; CONTROLLED STUDY; DYSTROGLYCANOPATHY; FEASIBILITY STUDY; FIBROSIS; GENE OVEREXPRESSION; IN VIVO STUDY; KNOCKOUT MOUSE; LIGAND BINDING; MOTOR PERFORMANCE; MOUSE; MUSCULAR DYSTROPHY; NECROSIS; NEWBORN; NONHUMAN; PHENOTYPE; SKELETAL MUSCLE;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DYSTROGLYCANS; FIBROSIS; GENETIC THERAPY; GENETIC VECTORS; GLYCOSYLATION; MICE; MICE, TRANSGENIC; MOTOR ACTIVITY; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; N-ACETYLGLUCOSAMINYLTRANSFERASES; PHENOTYPE; TRANSDUCTION, GENETIC;

MUS;

EID: 84875465831     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2012.084     Document Type: Article

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