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 代理人
主权项
地址