Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice

Molecular Therapy

Volumn 25, Issue 4, 2017, Pages 855-869

  Pozsgai, Eric R ^a,b   Griffin, Danielle A ^b   Heller, Kristin N ^b   Mendell, Jerry R ^a,b   Rodino Klapac, Louise R ^a,b  

^a OHIO STATE UNIVERSITY   (United States)

^b THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

Author keywords

AAV; gene therapy; LGMD2E; limb girdle muscular dystrophy; muscular dystrophy; sarcoglycan

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; BETA SARCOGLYCAN; CREATINE KINASE; SARCOGLYCAN; SGCB PROTEIN, HUMAN;

ADVERSE DRUG REACTION; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CARDIOMYOPATHY; CODON; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; DIAPHRAGM; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG SAFETY; FUNCTIONAL DISEASE; GENE CONSTRUCT; GENE EXPRESSION; GENE TRANSFER; HISTOPATHOLOGY; KYPHOSCOLIOSIS; LIMB GIRDLE MUSCULAR DYSTROPHY; LIMB-GIRDLE MUSCULAR DYSTROPHY TYPE 2E; MOUSE; MUSCLE STRENGTH; MYOPATHY; NONHUMAN; PROMOTER REGION; SGCB GENE; TRANSGENE; TREATMENT RESPONSE; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT; ADMINISTRATION AND DOSAGE; ANIMAL; BIOPSY; CARDIAC MUSCLE; CARDIOMYOPATHIES; CONVALESCENCE; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE ORDER; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HUMAN; KNOCKOUT MOUSE; KYPHOSIS; METABOLISM; MICRO-COMPUTED TOMOGRAPHY; MOTOR ACTIVITY; PATHOLOGY; PATHOPHYSIOLOGY; PHARMACOKINETICS; SARCOGLYCANOPATHIES; SCOLIOSIS; SKELETAL MUSCLE; TISSUE DISTRIBUTION;

ANIMALS; BIOPSY; CARDIOMYOPATHIES; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE ORDER; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; KYPHOSIS; MICE; MICE, KNOCKOUT; MOTOR ACTIVITY; MUSCLE, SKELETAL; MYOCARDIUM; RECOVERY OF FUNCTION; SARCOGLYCANOPATHIES; SARCOGLYCANS; SCOLIOSIS; TISSUE DISTRIBUTION; TRANSDUCTION, GENETIC; X-RAY MICROTOMOGRAPHY;

EID: 85016993854     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.02.013     Document Type: Article

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