发明名称 |
RNAi modulation of SCAP and therapeutic uses thereof |
摘要 |
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a SCAP gene (Human SCAP gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a SCAP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Human SCAP expression and the expression of a SCAP gene using the pharmaceutical composition; and methods for inhibiting the expression of a SCAP gene in a cell.
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申请公布号 |
US8383805(B2) |
申请公布日期 |
2013.02.26 |
申请号 |
US201113080334 |
申请日期 |
2011.04.05 |
申请人 |
ALNYLAM PHARMACEUTICALS, INC.;BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM;SOUTSCHEK JUERGEN;TAN PAMELA;HORTON JAY D.;BROWN MICHAEL S.;GOLDSTEIN JOSEPH L.;MOON YOUNG-AH |
发明人 |
SOUTSCHEK JUERGEN;TAN PAMELA;HORTON JAY D.;BROWN MICHAEL S.;GOLDSTEIN JOSEPH L.;MOON YOUNG-AH |
分类号 |
C07H21/04;C07H21/02;C12N15/113;C12N15/63;C12N15/85 |
主分类号 |
C07H21/04 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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