Extracellular Vesicle-Encapsulated AAVs for Therapeutic Gene Delivery to the Heart.

Xisheng Li,Sabrina La Salvia,Yaxuan Liang,Marta Adamiak,Erik Kohlbrenner, Dongtak Jeong, Elena Chepurko, Delaine Ceholski,Estrella Lopez-Gordo, Seonghun Yoon, Prabhu Mathiyalagan, Neha Agarwal, Divya Jha,Shweta Lodha, George Daaboul, Anh Phan,Nikhil Raisinghani,Shihong Zhang, Lior Zangi,Edgar Gonzalez Kozlova,Nicole Dubois,Navneet Dogra,Roger J Hajjar,Susmita Sahoo

CIRCULATION（2023）

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摘要

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.

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extracellular vesicles,genetic therapy

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