Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and eff...

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Autores principales: Hocquemiller, Michaël, Giersch, Laura, Audrain, Mickael, Parker, Samantha, Cartier, Nathalie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2016
Materias:
Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4960479/
https://www.ncbi.nlm.nih.gov/pubmed/27267688
http://dx.doi.org/10.1089/hum.2016.087
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author Hocquemiller, Michaël
Giersch, Laura
Audrain, Mickael
Parker, Samantha
Cartier, Nathalie
author_facet Hocquemiller, Michaël
Giersch, Laura
Audrain, Mickael
Parker, Samantha
Cartier, Nathalie
author_sort Hocquemiller, Michaël
collection PubMed
description Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and efficiency in transducing a wide range of cell types. The purpose of this review is to describe the most notable advancements in preclinical and clinical research on AAV-based CNS gene therapy and to discuss prospects for future development based on a new generation of vectors and delivery.
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spelling pubmed-49604792016-08-09 Adeno-Associated Virus-Based Gene Therapy for CNS Diseases Hocquemiller, Michaël Giersch, Laura Audrain, Mickael Parker, Samantha Cartier, Nathalie Hum Gene Ther Review Article Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and efficiency in transducing a wide range of cell types. The purpose of this review is to describe the most notable advancements in preclinical and clinical research on AAV-based CNS gene therapy and to discuss prospects for future development based on a new generation of vectors and delivery. Mary Ann Liebert, Inc. 2016-07-01 2016-06-07 /pmc/articles/PMC4960479/ /pubmed/27267688 http://dx.doi.org/10.1089/hum.2016.087 Text en © Michaël Hocquemiller, et al., 2016; Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons Attribution Noncommercial License (http://creativecommons.org/licenses/by-nc/4.0/) which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author(s) and the source are credited.
spellingShingle Review Article
Hocquemiller, Michaël
Giersch, Laura
Audrain, Mickael
Parker, Samantha
Cartier, Nathalie
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
title Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
title_full Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
title_fullStr Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
title_full_unstemmed Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
title_short Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
title_sort adeno-associated virus-based gene therapy for cns diseases
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4960479/
https://www.ncbi.nlm.nih.gov/pubmed/27267688
http://dx.doi.org/10.1089/hum.2016.087
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AT parkersamantha adenoassociatedvirusbasedgenetherapyforcnsdiseases
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