Non-viral gene therapy for Duchenne muscular dystrophy: Progress and challenges

Biochimica et Biophysica Acta - Molecular Basis of Disease

Volumn 1772, Issue 2, 2007, Pages 263-271

  Rando, Thomas A ^a,b  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b VA PALO ALTO HEALTH CARE SYSTEM   (United States)

Author keywords

Antisense oligonucleotide; Dystrophin; Gene editing; Muscular dystrophy; Plasmid gene therapy

Indexed keywords

2' O METHYLPHOSPHOROTHIOATE; ANTISENSE OLIGODEOXYNUCLEOTIDE; DYSTROPHIN; INTEGRASE; MORPHOLINE DERIVATIVE; MORPHOLINOPHOSPHORODIAMIDATE; OLIGODEOXYNUCLEOTIDE PHOSPHOROTHIOATE; OLIGONUCLEOTIDE; PLASMID DNA; PLASMID VECTOR; UNCLASSIFIED DRUG; VASCULOTROPIN;

ABSENCE OF SIDE EFFECTS; ANTISENSE THERAPY; CHILDHOOD; CLINICAL TRIAL; COST EFFECTIVENESS ANALYSIS; DISEASE MODEL; DRUG DELIVERY SYSTEM; DRUG DESIGN; DRUG EFFICACY; DRUG FORMULATION; DRUG SAFETY; DRUG STRUCTURE; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; GENE IDENTIFICATION; GENE TARGETING; GENE TRANSFER; HUMAN; METHODOLOGY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; OLIGONUCLEOTIDE THERAPY; PRIORITY JOURNAL; REVIEW; VIRAL GENE THERAPY;

ANIMALS; GENE THERAPY; GENETIC VECTORS; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE;

ANIMALIA;

EID: 33846419111     PISSN: 09254439     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.bbadis.2006.07.009     Document Type: Review

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