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Unlocking precision gene therapy: harnessing AAV tropism with nanobody swapping at capsid hotspots

1 Jul 2024

Abstract excerpt

Adeno-associated virus (AAV) has been remarkably successful in the clinic, but its broad tropism is a practical limitation of precision gene therapy. A promising path to engineer AAV tropism is the addition of binding domains to the AAV capsid that recognize cell surface markers present on a targeted cell type. We have recently identified two previously unexplored capsid regions near the 2/5-fold wall and 5-fold...

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PMID: 39022346
DOI: 10.1093/narmme/ugae008

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