发明名称 ENA NUCLEIC ACID PHARMACEUTICAL MODIFYING SPLICING IN MRNA PRECURSOR
摘要 <P>PROBLEM TO BE SOLVED: To provide a widely applicable and highly effective therapeutic agent by improving an antisense oligonucleotide to a splicing enhancer sequence (SES) present in exon 19, 41, 45, 46, 44, 50, 55, 51 or 53 of a dystrophin gene. <P>SOLUTION: There are provided oligonucleotides having a base sequence complementary to a specific nucleotide of a dystrophin cDNA (Gene Bank accession No. NM_004006.1), and a muscular dystrophy therapeutic agent containing the same. <P>COPYRIGHT: (C)2012,JPO&INPIT
申请公布号 JP2012135314(A) 申请公布日期 2012.07.19
申请号 JP20120027325 申请日期 2012.02.10
申请人 MATSUO MASAFUMI;TAKESHIMA YASUHIRO;NPO TRANSLATIONAL RESEARCH ORGANIZATION OF DUCHENNE MUSCULAR DYSTROPHY;KOBE TENNENBUTSU KAGAKU KK 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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