Rna interference mediating small rna molecules

C - Chemistry – Metallurgy – 07 – H

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C07H 21/02 (2006.01) A01K 67/033 (2006.01) A61K 31/712 (2006.01) A61K 48/00 (2006.01) A61P 37/00 (2006.01) C12N 5/10 (2006.01) C12N 15/11 (2006.01) C12P 19/34 (2006.01) C12Q 1/00 (2006.01) C12Q 1/68 (2006.01)

Patent

CA 2429814

Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a Drosophila in vitro system, we demonstrate that 19-23 nt short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs (siRNAs) are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex.

L'ARN bicaténaire (dsRNA) induit le silençage de gènes spécifiques d'une séquence lors de la maturation dans plusieurs organismes par un processus connu sous le nom d'interférence ARN (RNAi). Grâce à un système in vitro de drosophiles, nous pouvons démontrer que des fragments d'ARN courts 19-23 nt constituent les médiateurs spécifiques d'une séquence de RNAi. Les ARN interférants courts (siRNA) sont générés par une réaction de maturation de type RNase III à partir de dsRNA longs. Des doubles hélices de siRNA synthétisées chimiquement avec des extrémités 3' en porte à faux médient un clivage ARN cible efficace dans le lysat, et le site du clivage est situé près du centre de la région couverte par le siRNA guide. En outre, nous apportons la preuve que la direction de la maturation du dsRNA détermine si l'ARN cible sens ou anti-sens peut être clivé par le complexe siRNP produit.

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