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An adeno-associated virus variant enabling efficient ocular-directed gene delivery across species

6 May 2024

Abstract excerpt

Recombinant adeno-associated viruses (rAAVs) have emerged as promising gene therapy vectors due to their proven efficacy and safety in clinical applications. In non-human primates (NHPs), rAAVs are administered via suprachoroidal injection at a higher dose. However, high doses of rAAVs tend to increase additional safety risks. Here, we present a novel AAV capsid (AAVv128), which exhibits significantly enhanced...

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PMID: 38710714
DOI: 10.1038/s41467-024-48221-4

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