Current challenges and future Directions in recombinant AAV-mediated gene therapy of duchenne muscular dystrophy

Pharmaceuticals

Volumn 6, Issue 7, 2013, Pages 813-836

  Okada, Takashi ^a   Takeda, Shin'ichi ^a  

^a NATIONAL INSTITUTE OF NEUROSCIENCE   (Japan)

Author keywords

AAV; DMD; Immune response

Indexed keywords

ALIPOGENE TIPARVOVEC; DEPSIPEPTIDE; DYSTROPHIN; MESSENGER RNA; MICRODYSTROPHIN; MICROUTROPHIN; PARVOVIRUS VECTOR; PLACEBO; RECOMBINANT PARVOVIRUS VECTOR; REGULATOR PROTEIN; SMT C 1100; UNCLASSIFIED DRUG; UTROPHIN;

BIOTECHNOLOGICAL PROCEDURES; CELL MEDIATED CYTOTOXICITY; DNA MODIFICATION; DRUG APPROVAL; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE; GENE EXPRESSION; GENE REPLACEMENT THERAPY; GENETIC TRANSDUCTION; GENOME SIZE; HEART DISEASE; HUMAN; HYPERLIPOPROTEINEMIA TYPE 1; IMMUNE RESPONSE; IMMUNOREGULATION; LARGE SCALE PRODUCTION; LIMB PERFUSION; MESENCHYMAL STEM CELL; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; RNA SPLICING; STEM CELL GENE THERAPY; SURROGATE GENE; TRANSGENE; VECTOR PRODUCTION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIROGENESIS; VIRUS CAPSID; VIRUS DNA CELL DNA INTERACTION; VIRUS STRAIN;

EID: 84879820086     PISSN: None     EISSN: 14248247     Source Type: Journal    
DOI: 10.3390/ph6070813     Document Type: Review

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