Neurological diseases that result from an underlying genetic mutation or from acquired genetic and/or epigenetic changes are prevalent in the population, and might be amenable to gene therapy. In this Review, Simonato et al. discuss new developments in gene therapy vector design and delivery for the treatment of neurological diseases, including sensorineural disorders, Parkinson disease and epilepsy. The authors also outline the most promising therapeutic approaches to date.
- Michele Simonato
- Jean Bennett
- Joseph C. Glorioso