Gene therapy for neurological disorders: Progress and prospects

Benjamin E. Deverman, Bernard M. Ravina, Krystof S. Bankiewicz, Steven M. Paul, Dinah W.Y. Sah

Research output: Contribution to journalReview articlepeer-review

216 Scopus citations


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.

Original languageEnglish
Pages (from-to)641-659
Number of pages19
JournalNature Reviews Drug Discovery
Issue number9
StatePublished - Sep 1 2018


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