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Mol Ther Methods Clin Dev. 2019 Nov 26;17:69-82. doi: 10.1016/j.omtm.2019.11.012. eCollection 2020 Jun 12.

Adeno-Associated Viral Vectors in Neuroscience Research.

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Department of Pharmacology & Toxicology, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Medical Scientist Training Program, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Stark Neurosciences Research Institute, Indianapolis, IN 46202, USA.


Adeno-associated viral vectors (AAVs) are increasingly useful preclinical tools in neuroscience research studies for interrogating cellular and neurocircuit functions and mapping brain connectivity. Clinically, AAVs are showing increasing promise as viable candidates for treating multiple neurological diseases. Here, we briefly review the utility of AAVs in mapping neurocircuits, manipulating neuronal function and gene expression, and activity labeling in preclinical research studies as well as AAV-based gene therapies for diseases of the nervous system. This review highlights the vast potential that AAVs have for transformative research and therapeutics in the neurosciences.

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