完成1型糖尿病干细胞治疗的漫长征程。

Hunault Lise, Hesselson Daniel

Centenary Institute and Faculty of Medicine and Health, The University of Sydney, Sydney, NSW, 2006, Australia.

NPJ Metab Health Dis. 2024 Jul 22;2(1):9. doi: 10.1038/s44324-024-00014-5.

For over two decades pluripotent stem cells have promised a renewable source of β cells to treat patients with type 1 diabetes. Major efforts to optimize the differentiation, survival, and function of transplanted stem cell-derived tissue have recently delivered clinically meaningful metabolic benefits using a perforated encapsulation device that promotes integration with recipient vasculature under the protection of systemic immunosuppression. Despite this success, the journey is not over as a universal cure will require a larger β cell mass. Here, we summarize recent interdisciplinary advances that could maximize the functional β cell mass within transplanted devices and provide an immune privileged niche that could eliminate the need for systemic immunosuppression.

二十多年来，多能干细胞一直有望成为治疗1型糖尿病患者的可再生β细胞来源。最近，为优化移植的干细胞衍生组织的分化、存活和功能所做的重大努力，使用了一种多孔封装装置，在全身免疫抑制的保护下促进与受体血管系统的整合，从而带来了具有临床意义的代谢益处。尽管取得了这一成功，但旅程尚未结束，因为通用疗法需要更大的β细胞量。在这里，我们总结了最近的跨学科进展，这些进展可以使移植装置内的功能性β细胞量最大化，并提供一个免疫特权微环境，从而无需全身免疫抑制。