Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.
David M MarkusicTimothy C NicholsElizabeth P MerricksBrett PalaschakIrene ZolotukhinDamien MarsicSergei ZolotukhinArun SrivastavaRoland W HerzogPublished in: Journal of translational medicine (2017)
Several alternative strategies for capsid modification improve the in vivo performance of AAV vectors in hepatic gene transfer for correction of hemophilia. However, capsid optimization solely in mouse liver may not predict efficacy in other species and thus is of limited translational utility.