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Published July 2022 | Published + Supplemental Material
Journal Article Open

Adeno-Associated Virus Toolkit to Target Diverse Brain Cells


Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles for neuroscience research. They have two engineerable features: the capsid (outer protein shell) and cargo (encapsulated genome). These features can be modified to enhance cell type or tissue tropism and control transgene expression, respectively. Several engineered AAV capsids with unique tropisms have been identified, including variants with enhanced central nervous system transduction, cell type specificity, and retrograde transport in neurons. Pairing these AAVs with modern gene regulatory elements and state-of-the-art reporter, sensor, and effector cargo enables highly specific transgene expression for anatomical and functional analyses of brain cells and circuits. Here, we discuss recent advances that provide a comprehensive (capsid and cargo) AAV toolkit for genetic access to molecularly defined brain cell types.

Additional Information

© 2022 by Challis et al. This work is licensed under a Creative Commons Attribution 4.0 International License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. First published as a Review in Advance on April 19, 2022. We thank Dr. David Brown for generating the starburst plots in Figure 2, Dr. Catherine Oikonomou for editing, and Dr. Collin Challis for comments and discussion. We apologize to scientists whose work we could not include due to space and citation limitations. This work was funded by grants from the National Institutes of Health (NIH Pioneer DP1OD025535 to V.G. and BRAIN Initiative Armamentarium UF1MH128336 to V.G. and T.F.M.) and by the Beckman Institute for CLARITY, Optogenetics and Vector Engineering Research (https://clover.caltech.edu) for technology development and dissemination (to V.G. and T.F.M.). The collaboration between T.S.O. and V.G. was enabled by a Leverhulme Visiting Professor Award. Disclosure Statement: The California Institute of Technology has filed and licensed patent applications for some of the work described in this manuscript, with S.R.K., X.C., and V.G. listed as inventors. V.G. is a cofounder and board member of Capsida Biotherapeutics, a fully integrated AAV engineering and gene therapy company. The remaining authors are not aware of any affiliations, memberships, funding, or financial holdings that might be perceived as affecting the objectivity of this review.

Attached Files

Published - annurev-neuro-111020-100834.pdf

Supplemental Material - NE45_Gradinaru_SupplementalLiteratureCited.pdf

Supplemental Material - NE45_Gradinaru_SupplementalTables.xlsx


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August 22, 2023
December 22, 2023