| 发明名称 |
Pharmaceutical composition for treatment of duchenne muscular dystrophy |
| 摘要 |
A therapeutic pharmaceutical composition for patients of Duchenne muscular dystrophy with entire loss of exon 20 in dystrophin mature mRNA is provided. The composition comprise as an active principle an antisense oligonucleotide consisting of a 20 to 50-nucleotide sequence against exon 19 of the dystrophin pre-mRNA.
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| 申请公布号 |
US6653466(B2) |
申请公布日期 |
2003.11.25 |
| 申请号 |
US20010917907 |
申请日期 |
2001.07.31 |
| 申请人 |
JCR PHARMACEUTICALS CO., LTD.;MATSUO MASAFUMI |
发明人 |
MATSUO MASAFUMI |
| 分类号 |
C12N15/09;A61K31/00;A61K31/70;A61K31/7088;A61K38/00;A61K45/00;A61K48/00;A61P21/00;C12N15/113;(IPC1-7):C07H21/04;C12Q1/68;C12P19/34 |
主分类号 |
C12N15/09 |
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