Strategy for suppressing the expression of an endogenous gene

Details Strategy for suppressing the expression of an endogenous gene Strategy for suppressing the expression of an endogenous gene

A - Human Necessities

61

K

A61K 48/00 (2006.01) A61K 31/70 (2006.01) A61K 38/00 (2006.01) C07K 14/47 (2006.01) C07K 14/78 (2006.01) C12N 15/11 (2006.01)

Patent

CA 2232738

The invention provides strategy for suppressing expression of an endogenous gene, wherein said strategy comprises providing suppression effectors able to bind to the non-coding regions of a gene to be suppressed, to prevent the functional expression thereof. The suppression effectors may be antisense nucleic acids, and the non-coding regions can include the transcribed but non-translated regions of a gene. The strategy can also introduce a replacement gene.

L'invention se rapporte à une stratégie permettant de supprimer l'expression d'un gène endogène, ladite stratégie consistant à utiliser des effecteurs de suppression capables de se lier aux régions non codantes d'un gène à supprimer, de façon à éviter l'expression fonctionnelle dudit gène. Ces effecteurs de suppression peuvent être des acides nucléiques anti-sens et les régions non codantes peuvent inclure les régions transcrites mais non traduites d'un gène. Cette stratégie peut également consister à introduire un gène de remplacement.

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