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：
HRP20200822
公开号：
HRP20200822T1
申请日:
2020.05.21
申请国别(地区):
HR
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).
