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Adeno-Associated Virus Toolkit to Target Diverse Brain Cells

Challis, Rosemary C. and Ravindra Kumar, Sripriya and Chen, Xinhong and Goertsen, David and Coughlin, Gerard M. and Hori, Acacia M. and Chuapoco, Miguel R. and Otis, Thomas S. and Miles, Timothy F. and Gradinaru, Viviana (2022) Adeno-Associated Virus Toolkit to Target Diverse Brain Cells. Annual Review of Neuroscience, 45 . pp. 447-469. ISSN 0147-006X. doi:10.1146/annurev-neuro-111020-100834.

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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.

Item Type:Article
Related URLs:
URLURL TypeDescription Information
Challis, Rosemary C.0000-0003-3086-6553
Ravindra Kumar, Sripriya0000-0001-6033-7631
Chen, Xinhong0000-0003-0408-0813
Goertsen, David0000-0001-7138-1697
Hori, Acacia M.0000-0001-5868-348X
Chuapoco, Miguel R.0000-0001-5397-996X
Miles, Timothy F.0000-0001-6591-3271
Gradinaru, Viviana0000-0001-5868-348X
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 ( 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.
Group:Tianqiao and Chrissy Chen Institute for Neuroscience
Funding AgencyGrant Number
Beckman Institute for CLARITY, Optogenetics and Vector Engineering ResearchUNSPECIFIED
Leverhulme TrustUNSPECIFIED
Subject Keywords:targeted brain delivery, systemic delivery, adeno-associated virus, AAV, viral capsid engineering, viral cargo
Record Number:CaltechAUTHORS:20220525-90416000
Persistent URL:
Official Citation:Adeno-Associated Virus Toolkit to Target Diverse Brain Cells. Rosemary C. Challis, Sripriya Ravindra Kumar, Xinhong Chen, David Goertsen, Gerard M. Coughlin, Acacia M. Hori, Miguel R. Chuapoco, Thomas S. Otis, Timothy F. Miles, Viviana Gradinaru. Annual Review of Neuroscience 2022 45:1, 447-469; DOI: 10.1146/annurev-neuro-111020-100834
Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:114913
Deposited By: George Porter
Deposited On:25 May 2022 22:58
Last Modified:15 Jul 2022 22:20

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