Article

Engineering novel AAV capsids by global de-targeting and subsequent muscle-specific tropism in mice and NHPs

2025-05-19

Abstract excerpt

Recombinant adeno-associated viral (rAAV) vectors are a potent tool, but their clinical application is restricted by insufficient target tissue transduction and liver toxicity. We employed a novel two-step engineering strategy to create novel rAAV capsids with global tissue de-targeting, then produced strong tissue-specific expression by adding a peptide sequence. We created a novel capsid, AAV.Zero1, with globall...

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Identifiers and source

Literature Corpus work: 69105701-a2c0-518f-86ba-533cfd9d30fe
DOI: 10.1101/2025.05.19.654800

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