C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/713 (2006.01) A61K 48/00 (2006.01) C07H 21/02 (2006.01) C12N 15/63 (2006.01) C12N 15/85 (2006.01)
Patent
CA 2652770
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.
L'invention concerne un acide ribonucléique double brin (ARNds) pour inhiber l'expression d'un gène Aha (gène Aha1), comprenant un brin anti-sens ayant une séquence nucléotidique qui est inférieure à 30 nucléotides de long, généralement 19-25 nucléotides de long, et qui est sensiblement complémentaire à au moins une partie d'un gène Aha. L'invention concerne également une composition pharmaceutique comprenant l'ARNds avec un excipient pharmaceutiquement acceptable ; des procédés de traitement des maladies causées par l'expression d'Aha1 et l'expression d'un gène Aha utilisant la composition pharmaceutique ; et des procédés pour inhiber l'expression d'un gène Aha dans une cellule.
Balch William
Bramlage Birgit
Constien Rainer
Tan Pamela
Vornlocher Hans-Peter
Alnylam Pharmaceuticals Inc.
Smart & Biggar
The Scripps Research Institute
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