The Johns Hopkins University;Ionis Pharmaceuticals, Inc.
发明人:
RIGO, Frank,BENNETT, C., Frank,VAN OUTRYVE D'YDEWALLE, Constantin,SUMNER, Charlotte, J.
申请号：
EP17814164
公开号：
EP3471779A4
申请日:
2017.06.16
申请国别(地区):
EP
年份:
2020
代理人:
摘要：
Certain embodiments are directed to methods and compounds for modulating expression of SMN. In certain embodiments at least two compounds are used: a first compound for inhibiting SMN-NAT and increasing expression of SMN, and a second compound for modulating the splicing of SMN. Such methods and compounds are useful for increasing expression of exon 7 containing SMN mRNA in cells and animals.
