Nucleic acid and amino acid sequences encoding high-level...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/12 (2006.01) A61K 38/37 (2006.01) A61P 7/04 (2006.01) C07K 14/755 (2006.01) C12N 15/63 (2006.01) C12P 21/02 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2461443

Methods and compositions are provided that allow for high-level expression of a factor VIII polypeptide. More specifically, methods and compositions are provided comprising nucleic acid and amino acid sequences comprising a modified factor VIII that result in high-level expression of the polypeptide. The methods and compositions of the invention find use in the treatment of factor VIII deficiency including, for example, hemophilia A.

L'invention concerne des procédés et des compositions qui permettent d'exprimer à haut rendement un polypeptide de facteur VIII. Plus précisément, L'invention concerne des procédés et des compositions comprenant des séquences d'acides nucléiques et d'acides aminés comportant un facteur VIII modifié qui permet d'obtenir une expression à haut rendement du polypeptide. Ces procédés et compositions peuvent servir dans le traitement de la déficience en facteur VIII comprenant, par exemple, l'hémophilie A.

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