发明名称 SERPINA1 SIRNAS: COMPOSITIONS OF MATTER AND METHODS OF TREATMENT
摘要 The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpinal gene, and methods of using such dsRNA compositions to inhibit expression of Serpinal. In one embodiment, an iRNA for inhibiting expression of a Serpinal gene includes at least two sequences that are complementary to each other. The iRNA includes a sense strand having a first sequence and an antisense strand having a second sequence. The antisense strand includes a nucleotide sequence that is substantially complementary to at least part of an mRNA encoding Serpinal, and the region of complementarity is 30 nucleotides or less, and at least 15 nucleotides in length. Generally, the iRNA is 19 to 24, e.g., 19 to 21 nucleotides in length.
申请公布号 US2014235693(A1) 申请公布日期 2014.08.21
申请号 US201214127782 申请日期 2012.06.22
申请人 Sehgal Alfica;Bumcrot David;Bettencourt Brian 发明人 Sehgal Alfica;Bumcrot David;Bettencourt Brian
分类号 C12N15/113 主分类号 C12N15/113
代理机构 代理人
主权项
地址 Cambridge MA US