Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy

Molecular Therapy

Volumn 20, Issue 6, 2012, Pages 1212-1221

  Goyenvalle, Aurélie ^a,b   Wright, Jordan ^a   Babbs, Arran ^a   Wilkins, Vivienne ^a   Garcia, Luis ^b   Davies, Kay E ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b SORBONNE UNIVERSITÉ   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; SMALL NUCLEAR RNA; U7 SMALL NUCLEAR RNA; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE; GENE DELETION; GENE TARGETING; GENETIC ENGINEERING; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MULTIEXON SKIPPING; NONHUMAN; OPEN READING FRAME; SEQUENCE ANALYSIS; U7 SMALL NUCLEAR RNA GENE; VIRAL GENE DELIVERY SYSTEM;

MUS;

EID: 84861915467     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2012.26     Document Type: Article

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