申请人:Alnylam Pharmaceuticals, Inc.
公开号:US20150247143A1
公开(公告)日:2015-09-03
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
该发明涉及iRNA,例如双链核糖核酸(dsRNA),针对补体成分C5基因的组合物,以及使用这种iRNA,例如dsRNA,组合物来抑制C5的表达并治疗患有补体成分C5相关疾病的受试者,例如周期性夜间血红蛋白尿。