Progress toward Gene Therapy for Duchenne Muscular Dystrophy

Molecular Therapy

Volumn 25, Issue 5, 2017, Pages 1125-1131

  Chamberlain, Joel R ^a   Chamberlain, Jeffrey S ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

Author keywords

AAV; dystrophin; gene therapy; mdx mice; microdystrophin; muscular dystrophy

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; DYSTROPHIN; DMD PROTEIN, HUMAN;

DMD GENE; DUCHENNE MUSCULAR DYSTROPHY; DYSTROPHIN GENE; GENE; GENE EDITING; GENE TARGETING; GENE THERAPY; HUMAN; IMMUNE RESPONSE; IMMUNOREACTIVITY; MOLECULARLY TARGETED THERAPY; MUSCLE TISSUE; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); REVIEW; ANIMAL; ANIMAL MUSCULAR DYSTROPHY; CHEMISTRY; DEPENDOPARVOVIRUS; DOG; GENE VECTOR; GENETICS; HISTORY; METABOLISM; MOUSE; MUTATION; PATHOLOGY; PROCEDURES; PROTEIN DOMAIN; SKELETAL MUSCLE; TRENDS; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

ANIMALS; DEPENDOVIRUS; DOGS; DYSTROPHIN; GENETIC THERAPY; GENETIC VECTORS; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; MICE; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; PROTEIN DOMAINS;

EID: 85017446297     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.02.019     Document Type: Review

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