Methods and compositions for gene therapy for the treatment...

Details Methods and compositions for gene therapy for the treatment... Methods and compositions for gene therapy for the treatment...

C - Chemistry, Metallurgy

12

N

C12N 15/861 (2006.01) A61K 31/7088 (2006.01) A61K 38/17 (2006.01) A61K 48/00 (2006.01) A61P 3/06 (2006.01) C07K 14/705 (2006.01) C12N 5/10 (2006.01) C12N 15/12 (2006.01)

Patent

CA 2213254

The invention provides a recombinant viral vector comprising the DNA of, or corresponding to, at least a portion of the genome of an adenovirus, which portion is capable of infecting a hepatic cell; and a human VLDL receptor gene operatively linked to regulatory sequences directing its expression. The vector is capable of expressing the normal VLDL receptor gene product in hepatic cells in vivo or in vitro. This viral vector is useful in the treatment of metabolic disorders caused by the accumulation of LDL in plasma, such as familial hypercholesterolemia or familial combined hyperlipidemia.

L'invention concerne un vecteur viral de recombinaison comprenant à l'ADN d'au moins une partie du génome d'un adénovirus ou correspondant à au moins cette partie, laquelle est capable d'infecter une cellule hépatique. Elle porte également sur un gène récepteur de VLDL lié de manière fonctionnelle à des séquences de régulation dirigeant son expression. Le vecteur est capable d'exprimer in vivo ou in vitro le produit génique du récepteur de VLDL normal dans les cellules hépatiques. Ce vecteur viral est utile dans le traitement de troubles du métabolisme induits par l'accumulation de LDL dans le plasma, tels que l'hypercholestérolémie familiale ou l'hyperlipidémie familiale combinée.

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