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Open AccessReview

Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches

1
Group of Experimental Biotherapy and Diagnostics, Institute for Regenerative Medicine, Sechenov First Moscow State Medical University, Moscow 119991, Russia
2
Department of Forensics, University of Criminal Investigation and Police Studies, Belgrade 11000, Serbia
3
Department of Biomedical Engineering, University of Alabama at Birmingham, Birmingham, AL 35294, USA
4
Institute for Regenerative Medicine, Sechenov First Moscow State Medical University, Moscow 119991, Russia
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Institute for Uronephrology and Reproductive Health, Sechenov First Moscow State Medical University, Moscow 119991, Russia
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Institute of Photonic Technologies, Research Center “Crystallography and Photonics”, Russian Academy of Sciences, Troitsk, Moscow 142190, Russia
7
Department of Polymers and Composites, N.N. Semenov Institute of Chemical Physics, Moscow 119991, Russia
8
Chemistry Department, Lomonosov Moscow State University, Moscow 119991, Russia
*
Author to whom correspondence should be addressed.
Viruses 2020, 12(4), 460; https://doi.org/10.3390/v12040460
Received: 25 February 2020 / Revised: 6 April 2020 / Accepted: 15 April 2020 / Published: 18 April 2020
(This article belongs to the Special Issue Advances in Parvovirus Research 2020)
Neurodegenerative diseases (NDDs) are most commonly found in adults and remain essentially incurable. Gene therapy using AAV vectors is a rapidly-growing field of experimental medicine that holds promise for the treatment of NDDs. To date, effective delivery of a therapeutic gene into target cells via AAV has been a major obstacle in the field. Ideally, transgenes should be delivered into the target cells specifically and efficiently, while promiscuous or off-target gene delivery should be minimized to avoid toxicity. In the pursuit of an ideal vehicle for NDD gene therapy, a broad variety of vector systems have been explored. Here we specifically outline the advantages of adeno-associated virus (AAV)-based vector systems for NDD therapy application. In contrast to many reviews on NDDs that can be found in the literature, this review is rather focused on AAV vector selection and their testing in experimental and preclinical NDD models. Preclinical and in vitro data reveal the strong potential of AAV for NDD-related diagnostics and therapeutic strategies. View Full-Text
Keywords: AAV; neuro-degenerative disease; gene therapy AAV; neuro-degenerative disease; gene therapy
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Mijanović, O.; Branković, A.; Borovjagin, A.V.; Butnaru, D.V.; Bezrukov, E.A.; Sukhanov, R.B.; Shpichka, A.; Timashev, P.; Ulasov, I. Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches. Viruses 2020, 12, 460.

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