Gene therapy for hemophilia a

Details Gene therapy for hemophilia a Gene therapy for hemophilia a

C - Chemistry, Metallurgy

12

N

C12N 15/62 (2006.01) A01K 67/027 (2006.01) A61K 38/36 (2006.01) A61K 38/37 (2006.01) C07K 14/745 (2006.01) C07K 14/755 (2006.01)

Patent

CA 2450125

Nucleic acid constructs comprising procoagulant gene sequences under the control of a megakaryocyte-specific promoter are provided. The sequences preferably also comprise a secretory granule sorting domain Also provided are vectors comprising the sequences and methods of gene therapy comprising the use of the various constructs.

L'invention concerne des produits de synth­se d'acide nucl~ique comprenant des s~quences g~niques procoagulantes sous le contrÙle d'un promoteur sp~cifique du m~gakaryocate. Les s~quences comprennent de pr~f~rence ~galement un domaine de tri de granules s~cr~toires. L'invention concerne en outre des vecteurs comprenant les s~quences et les m~thodes de th~rapie g~nique int~grant l'utilisation des diff~rents produits de synth­se.

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