Preclinical and clinical progress in hemophilia gene therapy. Vrije Universiteit Brussel
PURPOSE OF REVIEW: Hemophilia A and B are attractive target diseases for gene therapy, as stable expression of coagulation factor VIII and IX may correct the bleeding diathesis. This review focuses on the recent progress in preclinical and clinical studies in gene therapy for hemophilia A and B.
RECENT FINDINGS: Hepatic gene delivery using vectors derived from adeno-associated virus (AAV) resulted in therapeutic but transient ...
RECENT FINDINGS: Hepatic gene delivery using vectors derived from adeno-associated virus (AAV) resulted in therapeutic but transient ...