Clinical Advances in Gene Therapy Updates on Clinical Trials of Gene Therapy in Haemophilia
The Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy
Therapeutic hFIX Activity Achieved After Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs
Hyperactivity of Factor IX Padua (R338L) Depends on Factor VIIIa Cofactor Activity