Sequence-specific inhibition of small rna function

Details Sequence-specific inhibition of small rna function Sequence-specific inhibition of small rna function

C - Chemistry, Metallurgy

12

N

C12N 15/113 (2010.01) A61K 31/7088 (2006.01) A61P 25/28 (2006.01) A61P 35/00 (2006.01) C07H 21/00 (2006.01) C12N 15/00 (2006.01) C12N 15/63 (2006.01) C12Q 1/68 (2006.01)

Patent

CA 2546669

The present invention relates to the discovery of a method for inhibiting RNA silencing in a target sequence-specific manner. RNA silencing requires a set of conserved cellular factors to suppress expression of gene-encoded polypeptide. The invention provides compositions for sequence-specific inactivation of the RISC component of the RNA silencing pathway, and methods of use thereof. The RISC inactivators of the present invention enable a variety of methods for identifying and characterizing miRNAs and siRNAs, RISC- associated factors, and agents capable of modulating RNA silencing. Therapeutic methods and compositions incorporating RISC inactivators and therapeutic agents identified through use of RISC inactivators are also featured.

La présente invention concerne la mise au point d'une méthode permettant d'inhiber le silençage par l'ARN selon un mode séquence-spécifique cible. Le silençage par l'ARN requiert la présence d'un ensemble de facteurs cellulaires conservés propres a supprimer l'expression d'un polypeptide codé par gène. L'invention concerne des compositions capables d'inactiver de façon séquence-spécifique la composante RISC de la voie de silençage par l'ARN et leurs méthodes d'utilisation. Les inactivateurs de RISC de la présente invention rendent possible l'application de diverses méthodes d'identification et de caractérisation de microARN, de petit ARN interférent, de facteurs associés à RISC et d'agents capables de moduler le silençage par l'ARN. Sont également décrites des méthodes et des compositions thérapeutiques incorporant des inactivateurs de RISC et des agents thérapeutiques identifiés au moyen desdits inactivateurs.

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