Viral vector-mediated gene transfer for CNS disease.

IMPORTANCE OF THE FIELD: Gene therapy is a promising strategy for the treatment of many neurological disorders that currently lack effective treatment. Recent improvements in vectorology and vector engineering have improved overall safety and delivery of viral vectors. AREAS COVERED IN THIS REVIEW: This review discusses the current state of viral vector development and clinical use, as well as routes of delivery, and clinical trials for neurological disorders. WHAT THE READER WILL GAIN: Viral vectors may be delivered directly or remotely to the CNS, largely depending on the nature of the disease and the tropism of the vector. Nonetheless, delivery remains one of the major limitations of successful gene transfer to the CNS. TAKE HOME MESSAGE: Although the majority of clinical trials have centered on gene replacement and neuroprotection approaches, the field is advancing in the direction of neuromodulation, gene silencing and other newer strategies.