Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells from a Duchenne muscular dystrophy patient

Brain Dev. 2001 Dec;23(8):788-90. doi: 10.1016/s0387-7604(01)00326-6.

Authors

Y Takeshima¹, H Wada, M Yagi, Y Ishikawa, Y Ishikawa, R Minami, H Nakamura, M Matsuo

Affiliation

¹ Department of Pediatrics, Kobe University Graduate School of Medicine, 7-5-1 Kusunokicho, Chuoku, Kobe 650 0017, Japan.

PMID: 11720794
DOI: 10.1016/s0387-7604(01)00326-6

No abstract available

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Cells, Cultured
Child
DNA, Recombinant / genetics*
Dystrophin / biosynthesis
Dystrophin / genetics*
Enhancer Elements, Genetic / genetics*
Exons / genetics
Frameshift Mutation / genetics
Genetic Therapy / methods*
Humans
Male
Muscle, Skeletal / metabolism
Muscle, Skeletal / physiopathology
Muscular Dystrophy, Duchenne / genetics
Muscular Dystrophy, Duchenne / metabolism
Muscular Dystrophy, Duchenne / therapy*
Oligonucleotides, Antisense / therapeutic use*
RNA, Messenger / genetics

Substances

DNA, Recombinant
Dystrophin
Oligonucleotides, Antisense
RNA, Messenger