Method for expression of small antiviral rna molecules...

A - Human Necessities – 61 – K

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A61K 48/00 (2006.01) A61K 35/12 (2006.01) A61K 35/14 (2006.01) A61P 31/18 (2006.01) C12N 15/00 (2006.01) C12N 15/11 (2006.01) C12N 15/867 (2006.01)

Patent

CA 2457282

In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector. The methods can be used to express double stranded RNA complexes. Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell, that interfere with a viral life cycle by down regulating either the viral genome, a viral genome transcript, or a host cell that . In another aspect the invention provides methods for treating patients having suffering from infection, particularly infection with HIV.

Selon un de ses aspects, l'invention se rapporte à des procédés et à des compositions d'expression de petites molécules d'ARN à l'intérieur d'une cellule au moyen d'un vecteur rétroviral. Lesdits procédés peuvent être mis en oeuvre pour exprimer des complexes d'ARN double-brin. La présente invention permet d'exprimer à l'intérieur d'une cellule de courts fragments d'ARN interférants (siARN), qui perturbent le cycle de vie d'un virus par régulation négative soit du génome viral, soit d'un transcrit du génome viral, soit d'un gène de cellule hôte qui est nécessaire à la réplication virale. Selon un autre aspect, l'invention se rapporte à des méthodes de traitement de patients souffrant d'une infection, notamment de l'infection par le VIH.

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