Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Molecular Therapy

Volumn 26, Issue 10, 2018, Pages 2337-2356

  Duan, Dongsheng ^a,b,c  

^a University of Missouri   (United States)

^b UNIVERSITY OF MISSOURI   (United States)

^c UNIVERSITY OF MISSOURI   (United States)

Author keywords

AAV; adeno associated virus; Becker muscular dystrophy; BMD; clinical trial; DMD; Duchenne muscular dystrophy; dystrophin; micro dystrophin; systemic gene therapy

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; AMINOTRANSFERASE; DYSTROPHIN; MICRODYSTROPHIN; MUSCLE PROTEIN; MYOTUBULARIN; PF 06939926; SGT 001; SURVIVAL MOTOR NEURON PROTEIN 1; TROPONIN; UNCLASSIFIED DRUG;

AMINOTRANSFERASE BLOOD LEVEL; CENTRONUCLEAR MYOPATHY; DOSE RESPONSE; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; GENE CASSETTE; GENE EXPRESSION; GENE REPLACEMENT THERAPY; HUMAN; IN VIVO GENE TRANSFER; LOW DRUG DOSE; NONHUMAN; REVIEW; SIDE EFFECT; SYSTEMIC THERAPY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS CAPSID; WERDNIG HOFFMANN DISEASE; DEPENDOPARVOVIRUS; DRUG THERAPY; GENE THERAPY; GENE VECTOR; GENETICS; PATHOLOGY; SKELETAL MUSCLE;

DEPENDOVIRUS; DYSTROPHIN; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE;

EID: 85050958092     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2018.07.011     Document Type: Review

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