Adeno-associated Virus 9 mediated FKRP gene therapy restores functional glycosylation of α-dystroglycan and improves muscle functions

Molecular Therapy

Volumn 21, Issue 10, 2013, Pages 1832-1840

  Xu, Lei ^a   Lu, Pei Juan ^a   Wang, Chi Hsien ^b   Keramaris, Elizabeth ^a   Qiao, Chunping ^a   Xiao, Bin ^a   Blake, Derek J ^c   Xiao, Xiao ^b   Lu, Qi Long ^a  

^a LEVINE CANCER INSTITUTE   (United States)

^b University of North Carolina   (United States)

^c CARDIFF UNIVERSITY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS 9 VECTOR; ALPHA DYSTROGLYCAN; CREATINE KINASE; FUKUTIN RELATED PROTEIN; LEUCINE; PROLINE; UNCLASSIFIED DRUG; VIRUS VECTOR;

AMINO ACID SUBSTITUTION; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; DRUG EFFICACY; FKRP GENE; GENE EXPRESSION; GENE REPLACEMENT THERAPY; GOLGI COMPLEX; HEART MUSCLE; HEART MUSCLE CELL; KIDNEY PARENCHYMA; LIVER LEVEL; MISSENSE MUTATION; MOUSE; MOUSE MUTANT; MUSCLE FUNCTION; MUSCULAR DYSTROPHY; NONHUMAN; PROTEIN ANALYSIS; PROTEIN EXPRESSION; PROTEIN GLYCOSYLATION; PROTEIN LOCALIZATION; SINGLE DRUG DOSE; SKELETAL MUSCLE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 9;

ANIMALS; CELLS, CULTURED; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DYSTROGLYCANS; GENETIC THERAPY; GENETIC VECTORS; GLYCOSYLATION; INJECTIONS, INTRAPERITONEAL; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES, LIMB-GIRDLE; MUSCULAR DYSTROPHY, ANIMAL; PROTEINS; TRANSGENES;

EID: 84885021202     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.156     Document Type: Article

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