THERAPEUTIC AGENT FOR DUCHENNE MUSCULAR DYSTROPHY

摘要

PROBLEM TO BE SOLVED: To discover a new SES(Splicing Enhancer Sequence) in a dystrophin exon and to obtain a new therapeutic agent for Duchenne muscular dystrophy based on the SES. SOLUTION: This antisense oligonucleotide comprises a base sequence complementary to a base sequence of the formula agcaagaaga cagcagcauu gcaaag or ggaagcuaag gaagaagcug agcagg.