Gene therapy for hemophilia

doi:10.1097/01.moh.0000239700.94555.b1

Article

Gene therapy for hemophilia

2 Aug 2006

Abstract excerpt

PURPOSE OF REVIEW: This review will highlight the progress achieved in the past 2 years on using gene therapy to treat hemophilia in animals and humans. RECENT FINDINGS: There has been substantial progress in using gene therapy to treat animals with hemophilia. Novel approaches for hemophilia A in mice include expression of Factor VIII in blood cells or platelets derived from ex-vivo transduced hematopoietic stem...

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