发明名称 |
Reversible siRNA-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease |
摘要 |
Isolated double-stranded short interfering nucleic acid molecules inhibiting the expression of endogenous wild-type and exogenous human mutant huntintin genes in cells of a non-human mammal which are expressing both said huntingtin genes, and their application for the treatment of Huntington's disease as well as to study Huntington's disease in rodent models. |
申请公布号 |
US8217018(B2) |
申请公布日期 |
2012.07.10 |
申请号 |
US20080665137 |
申请日期 |
2008.06.18 |
申请人 |
PERRIN VALERIE;DEGLON NICOLE;COMMISSARIAT A L'ENERGIE ATOMIQUE ET AUX ENERGIESALTERNATIVES |
发明人 |
PERRIN VALERIE;DEGLON NICOLE |
分类号 |
A61K31/70;C07H21/02;C07H21/04;C12N5/00;C12N15/113;C12Q1/68 |
主分类号 |
A61K31/70 |
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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