Compositions and methods for inhibiting expression of factor...

Details Compositions and methods for inhibiting expression of factor... Compositions and methods for inhibiting expression of factor...

A - Human Necessities

61

K

A61K 48/00 (2006.01)

Patent

CA 2626690

The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor V Leiden mutant gene (Factor V Leiden mutant 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 Factor V Leiden mutant 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 Factor V Leiden mutant gene using the pharmaceutical composition; and methods for inhibiting the expression of the Factor V Leiden mutant gene gene in a cell.

La présente invention concerne un acide ribonucléique double brin (ARNdb) destiné à inhiber l'expression du gène mutant du Facteur V Leiden (gène mutant du Facteur V Leiden), comprenant un brin antisens présentant une séquence nucléotidique présentant une longueur inférieure à 25 nucléotides et étant substantiellement complémentaire à au moins une partie du gène mutant du Facteur V Leiden. L~invention concerne également une composition pharmaceutique comprenant l~ARNdb ainsi qu~un excipient pharmaceutiquement acceptable ; des procédés destinés à traiter des pathologies induites par l~expression du gène mutant du Facteur V Leiden en utilisant la composition pharmaceutique ; et des procédés destinés à inhiber l'expression du gène mutant du Facteur V Leiden dans une cellule.

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