National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
Mol Ther. 2021 Dec 1;29(12):3332-3344. doi: 10.1016/j.ymthe.2021.09.010. Epub 2021 Sep 20.
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.
美国国立神经病学与中风研究所（NINDS）于 2019 年 9 月在美国马里兰州贝塞斯达举行了一次题为“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。会议聚集了来自学术界、工业界、宣传和政府的多学科专家，他们在中枢神经系统靶向基因治疗领域拥有丰富的经验。该小组的任务是确定这一不断发展的领域中的关键挑战和差距，并提出潜在的解决方案。研讨会分为四个部分：（1）控制水平和位置，（2）提高传递和分布，（3）增强模型和制造，以及（4）影响患者。在研讨会之前，NINDS 为每个会议成立了由主要意见领袖（KOL）组成的工作组。在会前电话会议上，KOL 负责确定研究差距和关键障碍，这些障碍会延迟和/或阻止基因靶向疗法进入临床。这种方法使研讨会能够从解决问题的讨论和策略制定开始，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的策略，为中枢神经系统基因靶向治疗领域的社区提供信息，包括但不限于研究人员和资助者。
