Rna interference for the treatment of gain-of-function...

Details Rna interference for the treatment of gain-of-function... Rna interference for the treatment of gain-of-function...

A - Human Necessities

61

K

A61K 48/00 (2006.01) C07H 21/04 (2006.01)

Patent

CA 2579638

The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington~s disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.

La présente invention concerne la découverte d'un traitement efficace pour une variété de maladies à gain de fonction, et notamment la chorée de Huntington. En l'occurrence, on utilise la technologie ARNi contre des régions polymorphiques dans les gènes codant diverses protéines mutantes à gain de fonction, ce qui donne un traitement efficace pour la maladie à gain de fonction.

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