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Adeno-Associated Virus Toolkit to Target Diverse Brain Cells

Challis, Rosemary C; Kumar, Sripriya Ravindra; Chen, Xinhong; Goertsen, David; Coughlin, Gerard M; Hori, Acacia M; Chuapoco, Miguel R; ... Gradinaru, Viviana; + view all (2022) Adeno-Associated Virus Toolkit to Target Diverse Brain Cells. Annual Review of Neuroscience , 45 (1) pp. 447-469. 10.1146/annurev-neuro-111020-100834. Green open access

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Abstract

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.

Type: Article
Title: Adeno-Associated Virus Toolkit to Target Diverse Brain Cells
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1146/annurev-neuro-111020-100834
Publisher version: https://doi.org/10.1146/annurev-neuro-111020-10083...
Language: English
Additional information: This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third-party material in this article are included in the Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
Keywords: Science & Technology, Life Sciences & Biomedicine, Neurosciences, Neurosciences & Neurology, WIDESPREAD GENE-TRANSFER, CENTRAL-NERVOUS-SYSTEM, IN-VIVO, DIRECTED EVOLUTION, NONHUMAN-PRIMATES, THERAPY VECTOR, SEROTYPE 9, AAV, EXPRESSION, DELIVERY
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > The Sainsbury Wellcome Centre
URI: https://discovery.ucl.ac.uk/id/eprint/10175714
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