Factor IX

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

IL-15 blockade and rapamycin rescue multifactorial loss of factor VIII from AAV-transduced hepatocytes in hemophilia A mice

Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B

Gene Therapy for Coagulation Disorders

Liver Gene Therapy: Reliable and Durable?

How Close Are We to Achieving Durable and Efficacious Gene Therapy for Hemophilia A and B?

Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression

Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy

The Template-Jumping Editing Approach in F9-Associated Hemophilia B Gene Therapy

Long-term durability of rAAV gene therapy in hemophilia: Factor expression, clinical outcomes and underlying molecular mechanisms