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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.

SUBMITTER: Hocquemiller M

PROVIDER: S-EPMC4960479 | biostudies-literature | 2016 Jul

REPOSITORIES: biostudies-literature

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

Hocquemiller Michaël M Giersch Laura L Audrain Mickael M Parker Samantha S Cartier Nathalie N

Human gene therapy 20160701 7

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 dis ...[more]

PMID: 27267688

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