Language

English

Publication Date

9-14-2023

Journal

Molecular Therapy Methods & Clinical Development

DOI

37674904

PMID

PMC10477751

PMCID

PMC10477751

PubMedCentral® Posted Date

8-18-2023

PubMedCentral® Full Text Version

Post-print

Abstract

Recombinant adeno-associated viruses (rAAVs) have emerged as one of the most promising gene therapy vectors that have been successfully used in pre-clinical models of heart disease. However, this has not translated well to humans due to species differences in rAAV transduction efficiency. As a result, the search for human cardiotropic capsids is a major contemporary challenge. We used a capsid-shuffled rAAV library to perform directed evolution in human iPSC-derived cardiomyocytes (hiPSC-CMs). Five candidates emerged, with four presenting high sequence identity to AAV6, while a fifth divergent variant was related to AAV3b. Functional analysis of the variants was performed

Keywords

adeno-associated virus, cardiotropic, hiPSC-CM, cardiac organoids, cardiac slices

Published Open-Access

yes

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