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Nat Rev Drug Discov. 2018 Sep;17(9):641-659. doi: 10.1038/nrd.2018.110. Epub 2018 Aug 10.

Gene therapy for neurological disorders: progress and prospects.

Author information

1
Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, CA, USA.
2
Stanley Center for Psychiatric Research at Broad Institute, Cambridge, MA, USA.
3
Voyager Therapeutics, Cambridge, MA, USA.
4
Department of Neurological Surgery, University of California-San Francisco, San Francisco, CA, USA.

Abstract

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.

PMID:
30093643
DOI:
10.1038/nrd.2018.110

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