Article

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

2025-05-26

Abstract excerpt

<title>Abstract</title> <p>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 cap...

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Literature Corpus work: 381e2e08-87e7-5fb1-ab49-bc91d955047d
DOI: 10.21203/rs.3.rs-6674722/v1

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