Tian Zhi-Chen, Yin Xiao-Juan
1. Department of Pediatrics, Hainan Hospital of The PLA General Hospital, Sanya 572000, China.
2. Department of Pediatrics, Chinese PLA General Hospital, Beijing 100700, China.
Yi Chuan. 2023 Jan 20;45(1):42-51. doi: 10.16288/j.yczz.22-245.
The optimal diagnosis and treatment of pediatric diseases depend on more adequate understanding of pathophysiology. The advent of induced pluripotent stem cells (iPSCs) has provided new strategies for the research and therapy of pediatric diseases. iPSCs are pluripotent stem cells induced by reprogramming of mature cells. Now they can be induced from many types of somatic cells (such as fibroblasts, peripheral blood mononuclear cells and urine cells).With the improvement of various reprogramming methods, its generation procedure is more and more optimized, and the use of small molecules to induce iPSCs is one of the research focus now. Due to their ability to differentiate into a variety of cells, combined with the development of gene editing technology, iPSCs have been increasingly favored in the modeling of diseases and cell therapy, especially hereditary diseases, and have achieved some success in clinical treatment. But before they can be widely used in clinical treatment, there are still some problems to be solved, such as tumorigenicity, immunogenicity and heterogeneity. This article reviewed the source of iPSCs, reprogramming technology, applications of iPSCs in common childhood diseases, current problems and prospects, in order to deepen the understanding of iPSCs and provide reference for in-depth research in field of exploring mechanisms of diseases and therapy of diseases.
儿科疾病的最佳诊断和治疗取决于对病理生理学更充分的理解。诱导多能干细胞（iPSC）的出现为儿科疾病的研究和治疗提供了新策略。iPSC是通过对成熟细胞进行重编程诱导产生的多能干细胞。现在它们可以从多种体细胞（如成纤维细胞、外周血单核细胞和尿液细胞）诱导产生。随着各种重编程方法的改进，其生成过程越来越优化，利用小分子诱导iPSC是目前的研究热点之一。由于它们能够分化为多种细胞，结合基因编辑技术的发展，iPSC在疾病建模和细胞治疗，尤其是遗传性疾病方面越来越受到青睐，并在临床治疗中取得了一些成功。但在它们能够广泛应用于临床治疗之前，仍有一些问题需要解决，如致瘤性、免疫原性和异质性。本文综述了iPSC的来源、重编程技术、iPSC在常见儿童疾病中的应用、当前存在的问题及前景，以加深对iPSC的理解，并为深入研究疾病机制和疾病治疗领域提供参考。
