Extracellular Vesicle–Encapsulated Adeno-Associated Viruses for Therapeutic Gene Delivery to the Heart

202376 citationsJournal Articlehybrid Open Access

Authors

Xisheng Li · Icahn School of Medicine at Mount Sinai

Sabrina La Salvia · Anna Needs Neuroblastoma Answers

Yaxuan Liang · Beijing Normal University

Mateusz Adamiak · Icahn School of Medicine at Mount Sinai

Erik Kohlbrenner · Spark Therapeutics (United States)

Dongtak Jeong · Hanyang University

Elena Chepurko

Abstract

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.

Topics & Keywords

RNA Interference and Gene Delivery Extracellular vesicles in disease Virus-based gene therapy research

Publication Details

Published in: Circulation

Volume 148, Issue 5, pp. 405-425

DOI: 10.1161/circulationaha.122.063759

Field-Weighted Citation Impact: 11.68

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Extracellular Vesicle–Encapsulated Adeno-Associated Viruses for Therapeutic Gene Delivery to the Heart

Authors

Abstract

Topics & Keywords

Publication Details