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诱导型多能干细胞(induced pluripotent stem cell,i PSC)是干细胞治疗的重要手段。以过表达miR-302s制备i PSC的方法因兼备较高的诱导效率和较低的成瘤潜能,具有良好的应用前景。为综合评价体内miR-302s过表达的生物学效应以及且为临床转化提供实验依据,该研究设计构建了miR-302s可由药物诱导过表达的敲入小鼠模型。研究结果显示,在药物处理后,该模型小鼠可在体内持续稳定表达导入的miR-302s基因;初步表型分析发现,miR-302s的过表达可能导致个体脂蛋白和嘌呤代谢异常。该模型为在体研究miR-302s功能、探讨其用于干细胞治疗的可行性提供了有效工具。
Induced pluripotent stem cell (iPSC) is an important means of stem cell therapy. The method of preparing i PSC by overexpression of miR-302s has good application prospects due to both high induction efficiency and low tumorigenic potential. In order to comprehensively evaluate the biological effects of miR-302s overexpression in vivo and to provide an experimental basis for clinical transformation, a knock-in mouse model of miR-302s that can be overexpressed by drugs was designed and constructed. The results showed that after the drug treatment, the mouse model can consistently express the introduced miR-302s gene in vivo. Preliminary phenotypic analysis revealed that overexpression of miR-302s may lead to abnormal metabolism of lipoproteins and purines. The model provides an effective tool for studying the function of miR-302s in vivo and exploring its feasibility for stem cell therapy.