C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/113 (2010.01) A61K 31/7088 (2006.01) A61K 31/7115 (2006.01) A61K 31/712 (2006.01) A61K 31/7125 (2006.01) A61K 31/713 (2006.01)
Patent
CA 2731779
The invention relates to im-proved double-stranded RNAi constructs (sometimes referred to as "solo-rxRNA") and uses thereof. The construct comprises a struc-ture formed in some aspects of the invention by two identical single-stranded polynucleotides, with the structure having two double-stranded stem regions (each having less than 21 base pairs) and a loop or bulge having about 4 to 11 nucleotides on each strand. The construct is re-sistant to cleavage by Dicer or other Dicer-like RNase III enzymes and is capable of being loaded into a RISC complex to effect RNA in-terference. In addition, the nucleotides of the present hairpin constructs may be modified to greatly enhance functionality, such as stability and specificity.
L'invention porte sur des constructions d'ARNi double brin (parfois appelées « solo-rxARN ») et sur leurs utilisations. La construction comprend une structure formée dans certains aspects de l'invention par deux polynucléotides simple brin identiques, la structure comportant deux régions de branche double brin (comportant chacune moins de 21 paires de bases) et une boucle ou un renflement comportant environ 4 à 11 nucléotides sur chaque brin. La construction est résistante au clivage par Dicer ou d'autres enzymes Rnase III de type Dicer et est apte à être prise en charge par le complexe RISC pour effectuer une interférence d'ARN. De plus, les nucléotides des présentes constructions en épingle à cheveux peuvent être modifiés pour augmenter fortement la fonctionnalité, telle que la stabilité et la spécificité.
Kamens Joanne
Khvorova Anastasia
Pavco Pamela A.
Salomon William
Samarsky Dmitry
Rxi Pharmaceuticals Corporation
Smart & Biggar
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