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Article

A single amino acid variant in the variable region I of AAV capsid confers liver detargeting

2025-03-05

Abstract excerpt

<h4>ABSTRACT</h4> AAV capsid serotypes isolated from nature have been widely used in gene delivery and gene therapy. Recently, more than 1,000 distinct AAV capsids were identified from human clinical samples by high-throughput, long-read DNA sequencing. In this study, we tap into this broad natural biodiversity of AAV capsids to develop liver-tropic AAV capsids. We initially screened a subset of variants derived...

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Identifiers and source

Literature Corpus work
e5df9ff6-16a3-5def-955a-3653c5f215ba
DOI
10.1101/2025.03.04.641478
Open publication

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A single amino acid variant in the variable region I of AAV capsid confers liver detargetingDOI 10.1101/2025.03.04.641478
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