On May 11, 2020, the National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation (Gates Foundation) held an exploratory expert scientific roundtable to inform an NIH–Gates Foundation collaboration on the development of scalable, sustainable, and accessible HIV and sickle cell disease (SCD) therapies based on in vivo gene editing of hematopoietic stem cells (HSCs). A particular emphasis was on how such therapies could be developed for low-resource settings in sub-Saharan Africa. Paula Cannon, PhD, of the University of Southern California and Hans-Peter Kiem, MD, PhD, of the Fred Hutchinson Cancer Research Center served as roundtable cochairs. Welcoming remarks were provided by the leadership of NIH, NHLBI, and BMGF, who cited the importance of assessing the state of the science and charting a path toward finding safe, effective, and durable gene-based therapies for HIV and SCD. These remarks were followed by three sessions in which participants heard presentations on and discussed the therapeutic potential of modified HSCs, leveraging HSC biology and differentiation, and in vivo HSC targeting approaches. This roundtable serves as the beginning of an ongoing discussion among NIH, the Gates Foundation, research and patient communities, and the public at large. As this collaboration progresses, these communities will be engaged as we collectively navigate the complex scientific and ethical issues surrounding in vivo HSC targeting and editing. Summarized excerpts from each of the presentations are given hereunder, reflecting the individual views and perspectives of each presenter.

中文翻译：

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造血干细胞中安全有效的体内靶向和基因编辑：加速发展的策略

2020 年 5 月 11 日，美国国立卫生研究院 (NIH) 和比尔和梅琳达·盖茨基金会 (盖茨基金会) 举行了一次探索性专家科学圆桌会议，以告知 NIH 与盖茨基金会在开发可扩展、可持续和可获取的 HIV 方面的合作和基于体内的镰状细胞病 (SCD) 疗法造血干细胞（HSC）的基因编辑。特别强调如何为撒哈拉以南非洲的资源匮乏地区开发此类疗法。南加州大学的 Paula Cannon 博士和 Fred Hutchinson 癌症研究中心的医学博士 Hans-Peter Kiem 担任圆桌会议联合主席。NIH、NHLBI 和 BMGF 的领导发表了欢迎辞，他们提到了评估科学现状并为寻找安全、有效和持久的 HIV 和 SCD 基因疗法指明道路的重要性。这些评论之后是三个会议，参与者听取了关于改良 HSC、利用 HSC 生物学和分化以及在体内的治疗潜力的演讲和讨论HSC 靶向方法。本次圆桌会议是 NIH、盖茨基金会、研究和患者社区以及广大公众之间持续讨论的开始。随着这种合作的进展，这些社区将参与进来，因为我们共同解决围绕体内HSC 靶向和编辑的复杂科学和伦理问题。下面给出了每个演讲的摘要摘录，反映了每个演讲者的个人观点和观点。