Gene therapy for Duchenne muscular dystrophy

Expert Opinion on Orphan Drugs

Volumn 3, Issue 11, 2015, Pages 1255-1266

  Ramos, Julian ^a,b   Chamberlain, Jeffrey S ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Adeno associated viral vectors; Becker muscular dystrophy; Duchenne muscular dystrophy; Dystrophin; Gene therapy; Immune response; Micro dystrophin; Micro utrophin; Tolerization

Indexed keywords

ADENOVIRUS VECTOR; DYSTROPHIN;

BECKER MUSCULAR DYSTROPHY; CLINICAL TRIAL (TOPIC); DUCHENNE MUSCULAR DYSTROPHY; GENE REPLACEMENT THERAPY; GENE TRANSFER; HUMAN; IMMUNE RESPONSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN SYNTHESIS; REVIEW; TRANSGENE; VIRUS CAPSID;

EID: 84947492689     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2015.1088780     Document Type: Review

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