Retroviral gene therapy vectors and therapeutic methods...

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/86 (2006.01) A61K 38/19 (2006.01) A61K 38/37 (2006.01) A61K 38/46 (2006.01) A61K 38/49 (2006.01) A61L 27/38 (2006.01) C07K 14/755 (2006.01) C12N 9/72 (2006.01) C12N 9/78 (2006.01) C12N 15/867 (2006.01) A61K 35/12 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2191845

Retroviral vectors are disclosed which include an insertion site for genes of interest and are capable of expressing high levels of the protein derived from the genes of interest in a wide variety of transfected cell types. Also disclosed are retroviral vectors lacking a selectable marker, thus rendering them suitable for human gene therapy in the treatment of a variety of disease states without the co-expression of a marker product, such as an antibiotic. These retroviral vectors are especially suited for use in certain packaging cell lines.

La présente invention concerne des vecteurs rétroviraux comportant un site d'insertion pour des gènes concernés. Ces vecteurs rétroviraux sont capables d'exprimer, dans une grande diversité de types de cellules transfectées, des niveaux élevés de la protéine dérivée des gènes concernés. L'invention concerne également des vecteurs rétroviraux ne comportant pas de marqueur sélectionnable, ce qui fait qu'ils conviennent chez l'homme pour le traitement par thérapie génique de divers états de maladies sans co-expression d'un produit marqueur tel qu'un antibiotique. Ces vecteurs rétroviraux conviennent particulièrement à certaines lignées cellulaires d'assemblage.

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