ANTISENSE-INDUCED EXON EXCLUSION IN MYOSTATIN

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专利权人:

Sarepta Therapeutics; Inc.

发明人:

Frederick Joseph Schnell,Richard Keith Bestwick

申请号：

US15559257

公开号：

US20180355358A1

申请日:

2016.03.18

申请国别(地区):

US

年份:

2018

代理人:

摘要：

The present disclosure relates to antisense oligomers and related compositions and methods for decreasing the expression of functional human myostatin and methods for treating muscular dystrophy and related disorders and more specifically relates to inducing exclusion of myostatin exon 2 and thereby reducing the levels of myostatin protein.

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