Cold Spring Harbor Laboratory;Isis Pharmaceuticals, Inc.
发明人:
Brenda F. Baker,Adrian R. Krainer,Yimin Hua
申请号:
US13720474
公开号:
US08946183B2
申请日:
2012.12.19
申请国别(地区):
US
年份:
2015
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.