Compositions and methods for inhibiting expression of huntingtin gene
摘要
<p>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.</p>
申请公布号
EP2325315(A1)
申请公布日期
2011.05.25
申请号
EP20110000550
申请日期
2006.10.27
申请人
ALNYLAM PHARMACEUTICALS, INC.
发明人
SAH, DINAH, WEN-YEE;HADWIGER, PHILIPP;ROEHL, INGO;BRAMLAGE, BIRGIT;TAN, PAMELA;VORNLOCHER, HANS-PETER;BUMCROT, DAVID