Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy

Molecular Therapy

Volumn 17, Issue 7, 2009, Pages 1234-1240

  Goyenvalle, Aurélie ^a   Babbs, Arran ^a   van Ommen, Gert Jan B ^b   Garcia, Luis ^c   Davies, Kay E ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^c INSERM   (France)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; HETEROGENEOUS NUCLEAR RIBONUCLEOPROTEIN; HETEROGENEOUS NUCLEAR RIBONUCLEOPROTEIN A1; PARVOVIRUS VECTOR; RNA; SMALL NUCLEAR RNA; SMALL NUCLEAR RNA U7; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BINDING SITE; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; GENE EXPRESSION; GENE LOCUS; GENE MUTATION; GENE SILENCING; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MYOBLAST; NONHUMAN; OPEN READING FRAME; PROCESS OPTIMIZATION; PROTEIN BINDING; PROTEIN MOTIF; RNA SEQUENCE;

ANIMALS; BLOTTING, WESTERN; CELL LINE; CELLS, CULTURED; DYSTROPHIN; EXONS; GENE THERAPY; GENETIC VECTORS; HETEROGENEOUS-NUCLEAR RIBONUCLEOPROTEIN GROUP A-B; HUMANS; MICE; MICE, TRANSGENIC; MODELS, BIOLOGICAL; MUSCULAR DYSTROPHY, DUCHENNE; MYOBLASTS; NIH 3T3 CELLS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, SMALL NUCLEAR;

ADENO-ASSOCIATED VIRUS; MUS MUSCULUS;

EID: 67649849595     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.113     Document Type: Article

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