Journal
NATURE REVIEWS DRUG DISCOVERY
Volume 17, Issue 9, Pages 641-659Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nrd.2018.110
Keywords
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Funding
- Beckman Institute for the CLARITY, Optogenetics and Vector Engineering Research Center (CLOVER) at the California Institute of Technology
- Friedreich's Ataxia Research Alliance (FARA)
- FARA Australasia
- CHDI Foundation
- Stanley Center for Psychiatric Research at Broad Institute
- Michael J. Fox Foundation
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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.
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