发明名称 ENA NUCLEIC ACID MEDICINE THAT MODIFIES SPLICING OF mRNA PRECURSOR
摘要 PROBLEM TO BE SOLVED: To provide a therapeutic agent having a wider applicable range and higher effect by improving antisense oligonucleotide to an SES (splicing enhancer sequence) that exists in exons 19, 41, 45, 46, 44, 50, 55, 51 or 53 of a dystrophin gene.SOLUTION: There is provided an oligonucleotide having a base sequence complementary to nucleotide number 2571-2607, 2578-2592, 2571-2592, 2573-2592, 2578-2596, 2578-2601, 2575-2592 or the like of a dystrophin cDNA (Gene Bank accession No. NM_004006.1), and a muscular dystrophy therapeutic agent containing the same.SELECTED DRAWING: None
申请公布号 JP2016182122(A) 申请公布日期 2016.10.20
申请号 JP20160095419 申请日期 2016.05.11
申请人 MATSUO MASAFUMI;TAKESHIMA YASUHIRO;ORPHAN DISEASE TREATMENT INSTITUTE CO LTD;DAIICHI SANKYO CO LTD 发明人 MATSUO MASAFUMI;TAKESHIMA YASUHIRO;KOIZUMI MAKOTO
分类号 C12N15/113;A61K31/7088;A61K38/00;A61K48/00;A61P21/04;C07H21/04 主分类号 C12N15/113
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