Adenoviral vectors for treatment of hemophilia

Details Adenoviral vectors for treatment of hemophilia Adenoviral vectors for treatment of hemophilia

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 48/00 (2006.01) C07K 14/755 (2006.01) C12N 9/64 (2006.01) C12N 15/861 (2006.01)

Patent

CA 2162497

An adenoviral vector including at least one DNA sequence encoding a clotting factor, such as, for example, Factor VIII, or Factor IX. Such vectors may be administered to a host in an amount effective to treat hemophilia in the host. The vectors infect hepatocytes very efficiently, whereby the hepatocytes express the DNA sequence encoding the clotting factor.

L'invention concerne un vecteur adénoviral renfermant au moins une séquence d'ADN codant pour un facteur de coagulation, tel que par exemple le Facteur VIII ou le Facteur IX. De tels vecteurs peuvent être administrés à un hôte en une quantité suffisante pour traiter l'hémophilie chez l'hôte. Ces vecteurs infectent les hépatocytes très efficacement, grâce à quoi les hépatocytes expriment la séquence d'ADN codant pour le facteur de coagulation.

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