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Published July 2018 | public
Journal Article

Viral Strategies for Targeting the Central and Peripheral Nervous Systems


Recombinant viruses allow for targeted transgene expression in specific cell populations throughout the nervous system. The adeno-associated virus (AAV) is among the most commonly used viruses for neuroscience research. Recombinant AAVs (rAAVs) are highly versatile and can package most cargo composed of desired genes within the capsid's ∼5-kb carrying capacity. Numerous regulatory elements and intersectional strategies have been validated in rAAVs to enable cell type–specific expression. rAAVs can be delivered to specific neuronal populations or globally throughout the animal. The AAV capsids have natural cell type or tissue tropism and trafficking that can be modified for increased specificity. Here, we describe recently engineered AAV capsids and associated cargo that have extended the utility of AAVs in targeting molecularly defined neurons throughout the nervous system, which will further facilitate neuronal circuit interrogation and discovery.

Additional Information

© 2018 Annual Reviews. Review in Advance first posted on April 25, 2018. We thank members of V.G.'s group at Caltech for useful discussions and Dr. Ken Chan and Jounhong Ryan Cho for critical input on sections of the review. We also thank Dr. John Bedbrook for critical reading of the manuscript. V.G. is supported by the NIH New Innovator Award DP2NS087949, the Presidential Early Career Awards for Scientists and Engineers, Stimulating Peripheral Activity to Relieve Conditions program OT2OD023848-01, and R01AG047664. V.G. is a Heritage Medical Research Institute Investigator and director of the Center for Molecular and Cellular Neuroscience in the Tianqiao and Chrissy Chen Institute for Neuroscience at Caltech. B.E.D. is supported by the Beckman Institute for the CLARITY, Optogenetics and Vector Engineering Research Center (CLOVER) for technology development and broad dissemination (http://clover.caltech.edu), and Friedreich's Ataxia Research Alliance (FARA) and FARA Australasia. C.N.B. is funded by a Ruth L. Kirschstein National Research Service Award (F31MH102913). Disclosure statement: The California Institute of Technology has filed patent applications related to this work with B.E.D. and V.G. listed as inventors. V.G. has recent grant support from the US federal government [National Institutes of Health (NIH) and National Science Foundation] to further develop, apply, and disseminate these methods. All protocols, information on reagents, and other information regarding these methods are freely available from the authors and online and disseminated via free, hands-on training courses (http://clover.caltech.edu). B.E.D. and V.G. also receive research support from Voyager Therapeutics (which licensed some of the related intellectual property from Caltech); this support was not used in preparation of this manuscript or for the studies described therein. C.N.B. declares no financial interests.

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