Alnylam Pharmaceuticals, Inc.;Icahn School of Medicine at Mount Sinai
发明人:
BETTENCOURT, BRIAN,FITZGERALD, KEVIN,QUERBES, WILLIAM,DESNICK, ROBERT J.,YASUDA, MAKIKO
申请号：
PL14800179
公开号：
PL3052628T3
申请日:
2014.10.03
申请国别(地区):
PL
年份:
2020
代理人:
摘要：
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting expression of ALAS1, wherein said dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region of complementarity to an ALAS1 RNA transcript, which antisense strand comprises at least 22 contiguous nucleotides from the antisense sequence of UAAGAUGAGACACUCUUUCUGGU (SEQ ID NO: 4153) or UAAGAUGAGACACUCTUUCUGGU (SEQ ID NO: 4154).
