Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells from a Duchenne muscular dystrophy patient.
Takeshima
Y
Y
Department of Pediatrics, Kobe University Graduate School of Medicine, 7-5-1 Kusunokicho, Chuoku, Kobe 650 0017, Japan.
Wada
H
H
Yagi
M
M
Ishikawa
Y
Y
Ishikawa
Y
Y
Minami
R
R
Nakamura
H
H
Matsuo
M
M
D002478:Cells, Cultured; D002648:Child; D004274:DNA, Recombinant / Q000235:genetics*; D016189:Dystrophin / Q000096:biosynthesis / Q000235:genetics*; D004742:Enhancer Elements, Genetic / Q000235:genetics*; D005091:Exons / Q000235:genetics; D016368:Frameshift Mutation / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008297:Male; D018482:Muscle, Skeletal / Q000378:metabolism / Q000503:physiopathology; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*; D012333:RNA, Messenger / Q000235:genetics
D004274:DNA, Recombinant; D016189:Dystrophin; D016376:Oligonucleotides, Antisense; D012333:RNA, Messenger