A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 48/00 (2006.01) A61K 31/713 (2006.01) A61P 25/14 (2006.01) C12N 15/11 (2006.01) C12N 15/85 (2006.01) C07H 21/04 (2006.01)
Patent
CA 2627025
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
L'invention concerne un acide ribonucléique bicaténaire (dsRNA) destiné à inhiber l'expression du gène Huntingtine (gène HD) et comprenant un brin antisens renfermant une séquence nucléotidique dont la longueur est inférieure à 25 nucléotides et qui est sensiblement complémentaire à une partie au moins du gène HD. L'invention concerne également une composition pharmaceutique comprenant le dsRNA en combinaison avec un support pharmaceutiquement acceptable, des méthodes destinées à traiter des maladies causées par l'expression du gène HD, ou d'une forme mutante correspondante, au moyen de cette composition pharmaceutique, ainsi que des méthodes destinées à inhiber l'expression du gène huntingtine dans une cellule.
Bramlage Birgit
Bumcrot David
Hadwiger Philipp
Roehl Ingo
Sah Dinah Wen-Yee
Alnylam Pharmaceuticals Inc.
Smart & Biggar
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