Department of Molecular Biology, Faculty of Medical Sciences in Katowice, Medical University of Silesia, Katowice, Medykow 18 Street, 40-752 Katowice, Poland.
Centre of Polymer and Carbon Materials, Polish Academy of Sciences, M. Curie-Sklodowskiej 34, 41-819 Zabrze, Poland.
Biomolecules. 2021 May 7;11(5):699. doi: 10.3390/biom11050699.
Induced pluripotent stem cells (iPSCs) are defined as reprogrammed somatic cells exhibiting embryonic stem cell characteristics. Since their discovery in 2006, efforts have been made to utilize iPSCs in clinical settings. One of the promising fields of medicine, in which genetically patient-specific stem cells may prove themselves useful, is gene therapy. iPSCs technology holds potential in both creating models of genetic diseases and delivering therapeutic agents into the organism via auto-transplants, which reduces the risk of rejection compared to allotransplants. However, in order to safely administer genetically corrected stem cells into patients' tissues, efforts must be made to establish stably pluripotent stem cells and reduce the risk of insertional tumorigenesis. In order to achieve this, optimal reprogramming factors and vectors must be considered. Therefore, in this review, the molecular bases of reprogramming safe iPSCs for clinical applications and recent attempts to translate iPSCs technology into the clinical setting are discussed.
诱导多能干细胞（iPSCs）被定义为具有胚胎干细胞特征的重编程体细胞核细胞。自 2006 年发现以来，人们一直在努力将 iPSCs 应用于临床环境。在基因治疗这一极具前景的医学领域中，遗传患者特异性干细胞可能具有很大的应用潜力。iPSCs 技术在创建遗传疾病模型和通过自体移植将治疗剂递送至生物体中具有潜力，与同种异体移植相比，这降低了排斥的风险。然而，为了将经过基因修正的干细胞安全地递送至患者组织中，必须努力建立稳定的多能干细胞，并降低插入性肿瘤发生的风险。为了实现这一目标，必须考虑最佳的重编程因子和载体。因此，在这篇综述中，讨论了用于临床应用的安全 iPSCs 重编程的分子基础以及将 iPSCs 技术转化为临床应用的最新尝试。
