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

TAKESHIMA Y; WADA H; YAGI M; ISHIKAWA Y; ISHIKAWA Y; MINAMI R; NAKAMURA H; MATSUO M

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J-GLOBAL ID：200902106701009252 Reference number：02A0167180

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

Duchenne型筋ジストロフィー患者の筋肉細胞でのジストロフィン産生を引き起こす接合増強連鎖に対するオリゴヌクレオチド

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Material：
Volume： 23 Issue： 8 Page： 788-790 Publication year： Dec. 2001
JST Material Number： W0814A ISSN： 0387-7604 Document type： Article
Article type：解説 Country of issue： Netherlands (NLD) Language： ENGLISH (EN)

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Musculoskeletal diseases

Reference (12)：

MONACO, AP. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics. 1988, 2, 90-95
MATSUO, M. Duchenne/Becker muscular dystrophy : from molecular diagnosis to gene therapy. Brain Dev. 1996, 18, 167-172
WILTON, SD. Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides. Neuromusc Disord. 1999, 9, 330-338
MANN, CJ. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci USA. 2001, 98, 42-47
TAKESHIMA, Y. Modulation of in vitro splicing of the upstream intron by modifying an intra-exon sequence which is deleted from the dystrophin gene in dystrophin Kobe. J Clin Invest. 1995, 95, 515-520

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