Invention Grant
US08217018B2 Reversible siRNA-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease
有权
突变型和内源性野生型亨廷顿病基因的可逆siRNA基沉默及其在亨廷顿病中的应用
- Patent Title: Reversible siRNA-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease
- Patent Title (中): 突变型和内源性野生型亨廷顿病基因的可逆siRNA基沉默及其在亨廷顿病中的应用
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Application No.: US12665137Application Date: 2008-06-18
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Publication No.: US08217018B2Publication Date: 2012-07-10
- Inventor: Valérie Perrin , Nicole Deglon
- Applicant: Valérie Perrin , Nicole Deglon
- Applicant Address: FR Paris
- Assignee: Commissariat a l'Energie Atomique et aux Energies Alternatives
- Current Assignee: Commissariat a l'Energie Atomique et aux Energies Alternatives
- Current Assignee Address: FR Paris
- Agency: Morgan, Lewis & Bockius LLP
- Priority: EP07290751 20070618
- International Application: PCT/IB2008/002603 WO 20080618
- International Announcement: WO2009/007855 WO 20090115
- Main IPC: A61K31/70
- IPC: A61K31/70 ; C07H21/02 ; C07H21/04 ; C12N5/00 ; C12Q1/68
Abstract:
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.
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