申请人:Alnylam Pharmaceuticals, Inc.
公开号:US07605251B2
公开(公告)日:2009-10-20
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the proprotein convertase subtilisin kexin 9 (PCSK9) gene, comprising an antisense strand having a nucleotide sequence which is less than 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; and methods for treating diseases caused by PCSK9 gene expression by using the pharmaceutical composition.
该发明涉及一种双链核糖核酸(dsRNA),用于抑制前蛋白酶转化亚硫酸酯基肽酶9 (PCSK9) 基因的表达,包括具有核苷酸序列的反义链,其长度小于30个核苷酸,通常为19-25个核苷酸,并且与PCSK9基因的至少一部分基本互补。该发明还涉及包括dsRNA和药学上可接受的载体的药物组合物;以及使用该药物组合物治疗由PCSK9基因表达引起的疾病的方法。