Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Michaël Hocquemiller; Laura Giersch; Mickael Audrain; Samantha Parker; Nathalie Cartier

doi:10.1089/hum.2016.087

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Michaël Hocquemiller
, Laura Giersch
, Mickael Audrain
, Samantha Parker
, Nathalie Cartier

Research output: Contribution to journal › Review article › peer-review

255 Scopus citations

Abstract

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.

Original language	English
Pages (from-to)	478-496
Number of pages	19
Journal	Human Gene Therapy
Volume	27
Issue number	7
DOIs	https://doi.org/10.1089/hum.2016.087
State	Published - Jul 2016
Externally published	Yes

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10.1089/hum.2016.087

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title = "Adeno-Associated Virus-Based Gene Therapy for CNS Diseases",

abstract = "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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AU - Parker, Samantha

AU - Cartier, Nathalie

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AB - 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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Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Abstract

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