MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy

Gene Therapy

Volumn 20, Issue 3, 2013, Pages 274-282

  Barbash, I M ^a   Cecchini, S ^a   Faranesh, A Z ^a   Virag, T ^a   Li, L ^a   Yang, Y ^a   Hoyt, R F ^a   Kornegay, J N ^b   Bogan, J R ^b   Garcia, L ^c   Lederman, R J ^a   Kotin, R M ^a  

^a NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^b UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^c CNRS   (France)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; DYSTROPHIN; SMALL NUCLEAR RNA;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CARDIOMYOPATHY; CATHETER; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; EXPERIMENTAL DOG; FEMALE; FLUOROSCOPY; GENETIC TRANSDUCTION; GENOME; HISTOPATHOLOGY; IMMUNOHISTOLOGY; MALE; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; OPEN READING FRAME; PRIORITY JOURNAL; PROTEIN EXPRESSION; RNA ANALYSIS; SEROTYPE; WESTERN BLOTTING; X RAY;

ANIMALS; BASE SEQUENCE; BLOTTING, WESTERN; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DOGS; DYSTROPHIN; EXONS; FEMALE; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HUMANS; IMMUNOHISTOCHEMISTRY; MAGNETIC RESONANCE IMAGING; MALE; MOLECULAR SEQUENCE DATA; MUSCULAR DYSTROPHY, DUCHENNE; MYOCARDIUM; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, SMALL NUCLEAR; SF9 CELLS; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS;

EID: 84874786215     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2012.38     Document Type: Article

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