Methods and compositions for rna interference

C - Chemistry – Metallurgy – 07 – H

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

Patent

CA 2525619

The invention provides methods and compositions for gene silencing by RNA interference. In particular, the invention provides methods for gene silencing or RNA knockdown using small interfering RNAs (siRNAs) having partial sequence homology to its target gene. The invention also provides methods for identifying common and/or differential responses to a plurality of different siRNAs targeting a gene. The invention also provides methods for evaluating the relative activity of the two strands of an siRNA. The invention further provides methods of designing siRNAs for gene silencing. The invention further provides methods of using siRNAs as therapeutics for treatment of diseases.

L'invention concerne des procédés et compositions permettant le silençage génique par l'interférence d'ARN. L'invention concerne notamment des procédés de silençage génique ou d'inactivité d'ARN par un ARNs de faible interférence (siRNAs) ayant une séquence partielle homologue à son gène cible. L'invention concerne également des procédés d'identification de réponses communes et/ou différentielles à une pluralité de différents siRNAs ciblant un gène. L'invention concerne également des procédés permettant d'évaluer l'activité relative des deux brins d'un siRNA. L'invention concerne enfin des procédés de conception de siRNAs pour le silençage de gène. L'invention concerne des procédés d'utilisation de siRNAs comme thérapeutiques destinés au traitement de maladies.

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