Srinivasan Mirra, Thangaraj Santhosh Raja, Arzoun Hadia
Internal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Cureus. 2021 Dec 19;13(12):e20516. doi: 10.7759/cureus.20516. eCollection 2021 Dec.
Type 1 diabetes (T1D) is one of the most prevalent early-onset autoimmune diseases, and numerous treatment regimens have been developed over the years with a mainstay focus on insulin injections, infusions, and pumps. However, with the evolution of modern medicine in the recent decade, can gene therapy be a possible solution to prevent and even cure this autoimmune diabetes? In this review, the authors discuss the present-day advancements around the globe where gene therapy is implemented in different techniques to halt and even reverse T1D. The main focus of the final included studies for this review was to regenerate or preserve pancreatic β cells from other cell types in order to optimize insulin secretions in non-obese autoimmune diabetic patients. A literature search was done in various databases such as PubMed, ScienceDirect, and Google Scholar, and a final of eight studies were included. On the whole, the studies reviewed suggested favorable results of gene therapy, although these researches were done mainly in vitro or as animal studies. The application of different virus vector encoding gene transfer through transcription factors, mRNA electroporation, insulin-like growth factor gene expression as well as combination gene transfer concluded beneficial effects on normalizing insulin production, which could pave the path to perfecting gene therapy, and may even find a permanent cure for T1D in the near future.
1型糖尿病(T1D)是最常见的早发性自身免疫性疾病之一,多年来已开发出多种治疗方案,主要集中在胰岛素注射、输注和泵治疗。然而,随着近十年来现代医学的发展,基因治疗能否成为预防甚至治愈这种自身免疫性糖尿病的可能解决方案?在这篇综述中,作者讨论了全球范围内基因治疗的最新进展,即在不同技术中实施基因治疗以阻止甚至逆转T1D。本综述最终纳入研究的主要重点是从其他细胞类型中再生或保存胰腺β细胞,以优化非肥胖自身免疫性糖尿病患者的胰岛素分泌。在PubMed、ScienceDirect和谷歌学术等各种数据库中进行了文献检索,最终纳入了八项研究。总体而言,所综述的研究表明基因治疗有良好的结果,尽管这些研究主要是在体外或作为动物研究进行的。通过转录因子进行不同病毒载体编码基因转移、mRNA电穿孔、胰岛素样生长因子基因表达以及联合基因转移的应用,对胰岛素产生的正常化产生了有益影响,这可能为完善基因治疗铺平道路,甚至可能在不久的将来找到T1D的永久治愈方法。