Reversible sirna-based silencing of mutant and endogenous...

Details Reversible sirna-based silencing of mutant and endogenous... Reversible sirna-based silencing of mutant and endogenous...

C - Chemistry, Metallurgy

12

N

C12N 15/11 (2006.01)

Patent

CA 2690730

Isolated double-stranded short interfering nucleic acid molecules inhibiting the expression of endogenous wild-type and exogenous human mutant huntingtin 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.

La présente invention concerne des molécules d'acide nucléique interférent court bicaténaire isolé qui inhibent l'expression de gènes huntingtine mutants exogènes et endogènes de type sauvage dans les cellules d'un mammifère non humain qui expriment les deux types desdits gènes huntingtine, et leur application lors du traitement de la maladie de Huntington, ainsi que lors de l'étude de la maladie de Huntington sur des modèles rongeurs.

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