Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary.

ABSTRACT: The National Institute of Neurological Disorders and Stroke (NINDS) held a workshop titled "Next generation strategies for gene-targeted therapies of central nervous system (CNS) disorders" in September 2019 in Bethesda, MD, USA. The meeting brought together a multi-disciplinary group of experts in the field of CNS-directed gene-targeted therapy delivery from academia, industry, advocacy, and the government. The group was charged with identifying the key challenges and gaps in this evolving field, as well as suggesting potential solutions. The workshop was divided into four sessions: (1) control of level and location, (2) improving delivery and distribution, (3) enhancing models and manufacturing, and (4) impacting patients. Prior to the workshop, NINDS established working groups of key op

SUBMITTER: Morris JA 

PROVIDER: S-EPMC8636163 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature