Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression
Therapeutic Advances in Hematology
Published online on January 30, 2026
Abstract
Therapeutic Advances in Hematology, Volume 17, January-December 2026.
Adeno-associated virus (AAV) gene therapy is a promising approach for hemophilia, offering the potential for sustained therapeutic expression of coagulation factors. However, both variability and durability of transgene expression remain a challenge, ...
Adeno-associated virus (AAV) gene therapy is a promising approach for hemophilia, offering the potential for sustained therapeutic expression of coagulation factors. However, both variability and durability of transgene expression remain a challenge, ...