Extracellular Vesicle-Encapsulated AAVs for Therapeutic Gene Delivery to the Heart.
Xisheng LiSabrina La SalviaYaxuan LiangMarta AdamiakErik KohlbrennerDongtak JeongElena ChepurkoDelaine CeholskiEstrella Lopez-GordoSeonghun YoonPrabhu MathiyalaganNeha AgarwalDivya JhaShweta LodhaGeorge DaaboulAnh PhanNikhil RaisinghaniShihong ZhangLior ZangiEdgar G KozlovaNicole DuboisNavneet DograRoger J HajjarSusmita SahooPublished in: Circulation (2023)
Together, using 5 different in vitro and in vivo model systems, we demonstrate significantly higher potency and therapeutic efficacy of EV-AAV vectors compared with free AAVs in the presence of NAbs. These results establish the potential of EV-AAV vectors as a gene delivery tool to treat heart failure.