Division of Endocrinology, Metabolism and Lipid Research, Department of Medicine, Washington University School of Medicine in St. Louis, and Department of Biomedical Engineering, School of Engineering & Applied Science, Washington University in St. Louis, St. Louis, MO
Semma Therapeutics, Inc., Cambridge, MA
Diabetes. 2017 May;66(5):1111-1120. doi: 10.2337/db16-1406.
Development of stem cell technologies for cell replacement therapy has progressed rapidly in recent years. Diabetes has long been seen as one of the first applications for stem cell-derived cells because of the loss of only a single cell type-the insulin-producing β-cell. Recent reports have detailed strategies that overcome prior hurdles to generate functional β-like cells from human pluripotent stem cells in vitro, including from human induced pluripotent stem cells (hiPSCs). Even with this accomplishment, addressing immunological barriers to transplantation remains a major challenge for the field. The development of clinically relevant hiPSC derivation methods from patients and demonstration that these cells can be differentiated into β-like cells presents a new opportunity to treat diabetes without immunosuppression or immunoprotective encapsulation or with only targeted protection from autoimmunity. This review focuses on the current status in generating and transplanting autologous β-cells for diabetes cell therapy, highlighting the unique advantages and challenges of this approach.
近年来，用于细胞替代治疗的干细胞技术发展迅速。由于仅损失单一细胞类型——产生胰岛素的β细胞，糖尿病长期以来被视为干细胞衍生细胞的首批应用之一。最近的报告详细介绍了克服先前障碍的策略，可从人多能干细胞（包括人诱导多能干细胞）体外生成功能性β样细胞。即使取得了这一成就，解决移植的免疫障碍仍然是该领域的主要挑战。从患者中开发出具有临床相关性的 hiPSC 衍生方法，并证明这些细胞可分化为β样细胞，为无需免疫抑制、免疫保护包封或仅针对自身免疫进行靶向保护的糖尿病治疗提供了新机会。本文重点介绍了用于糖尿病细胞治疗的自体β细胞生成和移植的最新进展，强调了该方法的独特优势和挑战。
