Gene therapy and ASOs have the potential to offer new therapeutic opportunities, and possibly cures, to patients where few or no treatment options exist for their disease. The Forum on Neuroscience and Nervous System Disorders hosted a 1.5-day public workshop on April 23-24, 2019 that brought together experts and key stakeholders from academia, government, industry, and non-profit organizations and explored approaches for advancing the development of gene therapies for nervous system disorders, including approaches using RNA interference and antisense oligonucleotides.