The gene therapy journey for hemophilia: are we there yet?

Abstract excerpt

Since the isolation and characterization of the genes for FVIII and FIX some 30 years ago, a longstanding goal of the field has been development of successful gene therapy for the hemophilias. In a landmark study published in 2011, Nathwani et al demonstrated successful conversion of severe hemophilia B to mild or moderate disease in 6 adult males who underwent intravenous infusion of an adeno-associated viral...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to CAR-T cell therapy research
• Share to CRISPR and Genetic Engineering
• Share to Virus-based gene therapy research