Recombinant aav vectors for gene therapy of hemophilia a

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/86 (2006.01) C07K 14/755 (2006.01) C12N 7/01 (2006.01) C12N 15/12 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2329505

The instant invention provides methods and materials for expressing a polypeptide with factor VIII activity comprising administering an rAAV vector encoding a truncated version of human factor VIII, containing, for example, a 90 kD heavy chain of factor VIII fused to a light chain of factor VIII.

La présente invention concerne des procédés et des matériaux qui permettent d'exprimer un polypeptide présentant l'activité du facteur VIII. Les procédés consistent à administrer un vecteur de virus adéno-associé de recombinaison codant une version tronquée du facteur VIII humain qui contient par exemple une chaîne lourde à 90 kD du facteur VIII qui est fusionnée à une chaîne légère du facteur VIII.

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