Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

Human Gene Therapy

Volumn 18, Issue 9, 2007, Pages 798-810

  Arechavala Gomeza, V ^a   Graham, I R ^b   Popplewell, L J ^b   Adams, A M ^c   Aartsma Rus, A ^d   Kinali, M ^a   Morgan, J E ^a   Van Deutekom, J C ^d,e   Wilton, S D ^c   Dickson, G ^b   Muntoni, Francesco ^a,f  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY OF LONDON   (United Kingdom)

^c UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^d LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^e Prosensa BV   (Netherlands)

^f HAMMERSMITH HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL CULTURE; CHEMISTRY; CONCENTRATION RESPONSE; DRUG MECHANISM; DRUG SAFETY; DRUG TARGETING; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; EXPLANT; GENE MUTATION; GENE SEQUENCE; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VIVO STUDY; MUSCLE CELL; NONHUMAN; NUCLEOTIDE SEQUENCE; RNA SPLICING; TECHNIQUE; TRANSGENIC MOUSE; UNITED KINGDOM;

ALTERNATIVE SPLICING; ANIMALS; BASE SEQUENCE; BLOTTING, WESTERN; CELLS, CULTURED; DYSTROPHIN; EXONS; GENE TARGETING; HUMANS; MICE; MICE, TRANSGENIC; MOLECULAR SEQUENCE DATA; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; ORGAN CULTURE TECHNIQUES; REPRODUCIBILITY OF RESULTS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA PRECURSORS; RNA, MESSENGER;

ANIMALIA; MUS MUSCULUS;

EID: 34848904544     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2006.061     Document Type: Article

References (68)

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