Unlocking Precision Gene Therapy: Harnessing AAV Tropism with Nanobody Swapping at Capsid Hotspots

Abstract excerpt

<h4>ABSTRACT</h4> Adeno-associated virus 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-fold valley...

Topics

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• CAR-T cell therapy research
• CRISPR and Genetic Engineering
• Herpesvirus Infections and Treatments
• interferon and immune responses
• Viral gastroenteritis research and epidemiology
• Viral Infections and Immunology Research
• Viral Infectious Diseases and Gene Expression in Insects
• Virus-based gene therapy research

Identifiers and source

Literature Corpus work

13016c54-3ded-5f16-a33e-e4e757df17c8

DOI

10.1101/2024.03.27.587049