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Review
. 2016 Jul;27(7):478-96.
doi: 10.1089/hum.2016.087.

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Michaël Hocquemiller  1 , Laura Giersch  1 , Mickael Audrain  2   3   4 , Samantha Parker  1 , Nathalie Cartier  3   4
Affiliations
Review

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Michaël Hocquemiller et al. Hum Gene Ther. 2016 Jul.

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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Figures

<b>Figure 1.</b>
Figure 1.
Routes of administration targeting the CNS by direct injection into the parenchyma or by injection into the cerebrospinal fluid via the intracerebroventricular or intrathecal (cisternal or lumbar) route.
See this image and copyright information in PMC

Comment in

  • The Target's the Thing.
    Flotte TR. Flotte TR. Hum Gene Ther. 2016 Jul;27(7):477. doi: 10.1089/hum.2016.29030.trf. Hum Gene Ther. 2016. PMID: 27428657 No abstract available.

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