申请人:Sah Wen-Yee Dinah
公开号:US20070099860A1
公开(公告)日:2007-05-03
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
该发明涉及一种双链核糖核酸(dsRNA),用于抑制Huntingtin基因(HD基因)的表达,包括具有核苷酸序列的反义链,其长度小于25个核苷酸,并且与HD基因的至少一部分基本互补。该发明还涉及包括dsRNA和药用载体的药物组合物;使用该药物组合物治疗由HD基因的表达或其突变形式引起的疾病的方法;以及在细胞中抑制huntingtin基因表达的方法。