DOI: 10.1073/pnas.2302997120 ISSN:

Targeted evolution of adeno-associated virus capsids for systemic transgene delivery to microglia and tissue-resident macrophages

Adam Young, Bjoern Neumann, Michael Segel, Civia Zi-Yu Chen, Panagiotis Tourlomousis, Robin J. M. Franklin
  • Multidisciplinary
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Tissue macrophages, including microglia, are notoriously resistant to genetic manipulation. Here, we report the creation of Adeno-associated viruses (AAV) variants that efficiently and widely transduce microglia and tissue macrophages in vivo following intravenous delivery, with transgene expression of up to 80%. We use this technology to demonstrate manipulation of microglia gene expression and microglial ablation, thereby providing invaluable research tools for the study of these important cells.

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