Gene therapy for neurological disorders: progress and prospects

Benjamin E. Deverman, Bernard M. Ravina, Krystof S. Bankiewicz, Steven M. Paul & Dinah W. Y. Sah; Nature Reviews Drug Discovery, Published: 10 August 2018 doi:10.1038/nrd.2018.110

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.


Gene therapy for neurological disorders: progress and prospects