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 opinion leaders (KOLs) for each session. In pre-meeting teleconferences, KOLs were tasked with identifying the research gaps and key obstacles that delay and/or prevent gene-targeted therapies to move into the clinic. This approach allowed for the workshop to begin with problem-solving discussions and strategy development, as the key issues had been established. The overall purpose of the workshop was to consider knowledge gaps and potential strategies to inform the community around CNS gene-targeted therapies, including but not limited to researchers and funders.

美国国家神经疾病和中风研究所 (NINDS) 于 2019 年 9 月在美国马里兰州贝塞斯达举办了题为“中枢神经系统 (CNS) 疾病基因靶向治疗的下一代策略”的研讨会。会议汇集了来自学术界、工业界、宣传机构和政府的 CNS 定向基因靶向治疗领域的多学科专家小组。该小组负责确定这一不断发展的领域中的主要挑战和差距，并提出可能的解决方案。研讨会分为四个部分：(1) 水平和位置控制，(2) 改进交付和分配，(3) 增强模型和制造，以及 (4) 影响患者。在研讨会之前，NINDS 为每届会议建立了关键意见领袖 (KOL) 工作组。在会前电话会议中，KOL 的任务是确定延迟和/或阻止基因靶向疗法进入临床的研究差距和主要障碍。这种方法允许研讨会从解决问题的讨论和战略制定开始，因为关键问题已经确定。研讨会的总体目的是考虑知识差距和潜在策略，以告知社区 CNS 基因靶向治疗，包括但不限于研究人员和资助者。