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Gene therapy for neurological disorders: progress and prospects

Deverman, Benjamin E. and Ravina, Bernard M. and Bankiewicz, Krystof S. and Paul, Steven M. and Sah, Dinah W. Y. (2018) Gene therapy for neurological disorders: progress and prospects. Nature Reviews Drug Discovery, 17 (9). pp. 641-659. ISSN 1474-1776. doi:10.1038/nrd.2018.110.

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Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic proteins, microRNAs, antibodies or gene-editing machinery have been successfully delivered to the central nervous system with natural or engineered viral capsids via various routes of administration. Importantly, initial clinical studies have demonstrated encouraging safety and efficacy in diseases such as Parkinson disease and spinal muscular atrophy, as well as durability of transgene expression. Here, we discuss key considerations and challenges in the future design and development of therapeutic AAV vectors, highlighting the most promising targets and recent clinical advances.

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Deverman, Benjamin E.0000-0002-6223-9303
Additional Information:© 2018 Springer Nature Limited. Published: 10 August 2018. The authors thank their colleagues at Voyager Therapeutics for numerous discussions on adeno-associated viral (AAV) gene therapy for central nervous system (CNS) disorders and the Parkinson disease team and P. Larson for their work on the delivery of AAV2–aromatic-L-amino-acid decarboxylase (AADC) gene therapy in patients with Parkinson disease with intraparenchymal brain administration. The authors also thank E. Smith, W. Yen and M. Lawrence for assistance with the figures, tables and text, respectively. B.E.D. was supported by the Beckman Institute for the CLARITY, Optogenetics and Vector Engineering Research Center (CLOVER) at the California Institute of Technology, the Friedreich's Ataxia Research Alliance (FARA) and FARA Australasia and the CHDI Foundation and is currently supported by the Stanley Center for Psychiatric Research at Broad Institute. K.S.B. was supported by the Michael J. Fox Foundation. B.M.R., S.M.P. and D.W.Y.S. are currently employees of Voyager Therapeutics, a CNS gene therapy company working on AAV vectors for the treatment of severe neurological diseases. Competing interests: The authors declare competing financial interests in the form of funding from Voyager Therapeutics, employment by Voyager Therapeutics and/or personal financial interests in Voyager Therapeutics.
Errata:Details related to the copyright permissions for the images shown in Figure 3 have been added to the figure legend.
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Caltech Beckman InstituteUNSPECIFIED
Friedreich's Ataxia Research AllianceUNSPECIFIED
Broad Institute of MIT and HarvardUNSPECIFIED
Michael J. Fox FoundationUNSPECIFIED
Issue or Number:9
Record Number:CaltechAUTHORS:20180813-080718032
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Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:88776
Deposited By: Tony Diaz
Deposited On:13 Aug 2018 15:23
Last Modified:01 Jun 2023 22:53

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