Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?

Abstract excerpt

Gene transfer is a novel area of therapeutics in which the active agent is a nucleic acid rather than a protein or small molecule. As early as 1997, investigators reported long-term expression of therapeutic levels of factor IX using gene transfer techniques in hemophilia B mice, and similar data were thereafter reported in mice with hemophilia A. Efforts to translate these results to hemophilic dog models at...

Topics

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

• Share to Adenoviridae
• Share to Dependovirus
• Share to Gene Transfer Techniques