COLD SPRING HARBOR LABORATORY;Isis Pharmaceuticals, Inc.
发明人:
Baker, Brenda F.,Krainer, Adrian R.,HUA, Yimin
申请号：
HUE12188625
公开号：
HUE027486T2
申请日:
2006.06.23
申请国别(地区):
HU
年份:
2016
代理人:
摘要：
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
