Rna silencing compositions and methods for the treatment of...

C - Chemistry – Metallurgy – 12 – N

Patent

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C12N 15/11 (2006.01) A61K 31/7105 (2006.01) A61K 31/713 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) A61P 25/14 (2006.01) C12N 15/63 (2006.01) C12N 15/85 (2006.01) C07H 21/02 (2006.01) C12N 15/12 (2006.01)

Patent

CA 2662704

The present invention relates to the discovery of an effective treatment for a variety of Huntington's disease (HD). The present invention utilizes RNA silencing technology (e.g. RNAi) against single nucleotide polymorphisms (SNPs) in the Huntingtin (htt) gene encoding the dominant, gain-of-function mutant Huntington protein, thereby resulting in an effective treatment for the gain-of-function disease.

La présente invention concerne la découverte d'un traitement efficace contre diverses formes de la chorée de Huntington (HD). L'invention met en AEuvre la technique de silençage de l'ARN (p. ex. l'ARNi) contre les polymorphismes mononucléotidiques (SNP) dans le gène de Huntington (htt) codant la protéine de Huntington mutante dominante à gain de fonction, ce qui donne un traitement efficace contre cette maladie liée à un gain de fonction.

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