School of Medical and Molecular Biosciences and the Centre for Health Technologies, University of Technology Sydney, Sydney, NSW, Australia.
1] School of Medical and Molecular Biosciences and the Centre for Health Technologies, University of Technology Sydney, Sydney, NSW, Australia [2] Translational Cancer Research Group, University of Technology Sydney, Sydney, NSW, Australia.
Gene Ther. 2015 Jan;22(1):1-8. doi: 10.1038/gt.2014.93. Epub 2014 Oct 23.
Type 1 diabetes results from the autoimmune destruction of the insulin-producing pancreatic beta (β) cells. Patients with type 1 diabetes control their blood glucose levels using several daily injections of exogenous insulin; however, this does not eliminate the long-term complications of hyperglycaemia. Currently, the only clinically viable treatments for type 1 diabetes are whole pancreas and islet transplantation. As a result, there is an urgent need to develop alternative therapies. Recently, cell and gene therapy have shown promise as a potential cure for type 1 diabetes through the genetic engineering of 'artificial' β cells to regulate blood glucose levels without adverse side effects and the need for immunosuppression. This review compares putative target cells and the use of pancreatic transcription factors for gene modification, with the ultimate goal of engineering a glucose-responsive 'artificial' β cell that mimics the function of pancreatic β cells, while avoiding autoimmune destruction.
1 型糖尿病是由产生胰岛素的胰腺β（β）细胞的自身免疫性破坏引起的。1 型糖尿病患者通过每日多次注射外源性胰岛素来控制血糖水平；然而，这并不能消除高血糖的长期并发症。目前，1 型糖尿病的唯一临床可行治疗方法是全胰腺和胰岛移植。因此，迫切需要开发替代疗法。最近，细胞和基因治疗作为 1 型糖尿病的潜在治疗方法显示出了希望，通过对“人工”β细胞进行基因工程改造，使其在无需免疫抑制的情况下调节血糖水平，而没有不良反应。这篇综述比较了潜在的靶细胞和胰腺转录因子在基因修饰中的应用，最终目标是设计一种对葡萄糖有反应的“人工”β细胞，使其模拟胰腺β细胞的功能，同时避免自身免疫性破坏。
