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filingDate 2019-12-06-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationDate 2022-02-09-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber JP-2022513719-A
titleOfInvention Oligonucleotide composition and its method
abstract PROBLEM TO BE SOLVED: To provide a designed DMD oligonucleotide, a composition thereof and a method of use thereof. In some embodiments, the present disclosure skips exons 51 so that the transcript can be translated into a dystrophin protein variant that is internally truncated but at least partially functional. This provides a useful technique for repairing mutant DMD transcripts. In some embodiments, the present disclosure provides techniques useful for regulating DMD transcript splicing. In some embodiments, the techniques provided can alter the splicing of dystrophin (DMD) DMD transcripts. In some embodiments, the present disclosure provides methods of treating diseases such as muscular dystrophy, including, but not limited to, Duchenne muscular dystrophy, Becker muscular dystrophy and the like. [Selection diagram] Fig. 1
priorityDate 2018-12-06-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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