Advances in the application of induced pluripotent stem cells in pediatric diseases

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.

儿童疾病的最佳诊断和治疗依赖于对病理生理学更充分的认识,而诱导多能干细胞(induced pluripotent stem cells, iPSCs)的出现则为儿童疾病的研究和治疗提供了新的策略。iPSCs是由成熟细胞经重编程诱导而产生的具有多能性的干细胞,目前可从多种类型的体细胞(如成纤维细胞、外周血单个核细胞和尿液细胞等)诱导生成。其生成过程随着各种重编程方法的改进而越来越完善,其中利用小分子进行诱导是目前研究的热点。由于具有向多种细胞分化的能力,并且结合基因编辑技术的发展,目前它在模拟疾病和细胞治疗中的作用越来越受到青睐,特别是遗传性疾病,并且在临床治疗方面已经取得了一些成功。但在其广泛应用于临床治疗之前,仍存在一些问题需要解决,如致瘤性、免疫原性和异质性。本文重点对iPSCs来源、重编程技术、iPSCs在儿童常见疾病中的应用、目前存在的问题及展望等方面展开综述,以加深对iPSCs的理解,并为iPSCs在探索疾病的机制以及治疗领域的深入研究提供参考。.


cell therapy; disease modeling; induced pluripotent stem cells; reprogramming.

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