Author(s) :

Chang Ma, Jiayi Lu.

Volume/Issue :

Abstract :

Type 1 diabetes (T1D) is a chronic autoimmune disease affecting millions of people worldwide. The condition results from the immune-mediated destruction of pancreatic β-cells, leading to insulin deficiency and lifelong dependency on insulin therapy. Despite advances in insulin delivery and glucose monitoring technologies, many patients struggle to maintain optimal glycemic control, and severe hypoglycemia remains a persistent risk. Islet transplantation offers a potential alternative but is limited by donor shortages and the need for lifelong immunosuppression. Pluripotent stem cells (PSCs), including embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), offer a renewable and scalable source of insulin-producing β-cells. These cells can be differentiated in vitro and have demonstrated glucose-responsive insulin secretion and the ability to reverse diabetes in animal models. Recent years have seen the translation of PSC-derived β-cell therapies into clinical trials, with several promising candidates including VX-880, VC-02, CTX-211 and OZTx-410 under investigation. These approaches include both allogeneic and autologous strategies as well as gene-edited and encapsulated cell delivery systems designed to enhance cell survival and minimize immune rejection. This article reviews the latest advances in PSC-based cell therapies for T1D with a focus on differentiation protocols, preclinical studies and ongoing clinical trials. It also discusses current challenges including immune protection, vascular integration, and scalability and outlines future directions for achieving a functional cure through regenerative medicine.