Safe adeno-associated virus 2 vector for gene therapy

Details Safe adeno-associated virus 2 vector for gene therapy Safe adeno-associated virus 2 vector for gene therapy

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 31/70 (2006.01) A61K 35/28 (2006.01) A61K 48/00 (2006.01) C07K 14/805 (2006.01) C12N 5/10 (2006.01) C12N 15/11 (2006.01) C12N 15/864 (2006.01) C12N 15/90 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2098483

Gene therapy involves the transfer and stable insertion of new genetic information into cells. The present invention is directed to safe vectors for gene therapy and thus provides hybrid parvovirus vectors which are capable of site-specific integration into a mammalian chromosome without substantial cytotoxicity, and which direct erythroid cell-specific expression of heterologous genes. The hybrid vector is useful in gene therapy, particularly in the treatment of hemoglobinopathies and other hematopoietic diseases, and in conferring cell-specific multidrug resistance. A method of delivery of constitutive levels of a pharmaceutical product and a method of producing a recombinant protein are also provided.

La thérapie génétique implique le transfert et l'insertion stable d'informations génétiques nouvelles dans des cellules. L'invention concerne des vecteurs inoffensifs de thérapie génétique et fournit ainsi des vecteurs parvoviraux hybrides capables de s'intégrer dans des sites spécifiques d'un chromosome d'un mammifère sans aucune cytotoxicité significative et de diriger l'expression de gènes hétérologues spécifiques à des cellules érythroïdes. Ce vecteur hybride est utile en thérapie génétique, notamment pour traiter des hémoglobinopathies et d'autres maladies hématopoïétiques, et pour octroyer à des cellules spécifiques une résistance à des médicaments multiples. L'invention concerne en outre un procédé d'administration de niveaux constitutifs d'un produit pharmaceutique et un procédé de production d'une protéine de recombinaison.

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