Lyssiotis Costas A, Foreman Ruth K, Staerk Judith, Garcia Michael, Mathur Divya, Markoulaki Styliani, Hanna Jacob, Lairson Luke L, Charette Bradley D, Bouchez Laure C, Bollong Michael, Kunick Conrad, Brinker Achim, Cho Charles Y, Schultz Peter G, Jaenisch Rudolf
Department of Chemistry, Scripps Research Institute, La Jolla, CA 92037, USA.
Proc Natl Acad Sci U S A. 2009 Jun 2;106(22):8912-7. doi: 10.1073/pnas.0903860106. Epub 2009 May 15.
Ectopic expression of defined transcription factors can reprogram somatic cells to induced pluripotent stem (iPS) cells, but the utility of iPS cells is hampered by the use of viral delivery systems. Small molecules offer an alternative to replace virally transduced transcription factors with chemical signaling cues responsible for reprogramming. In this report we describe a small-molecule screening platform applied to identify compounds that functionally replace the reprogramming factor Klf4. A series of small-molecule scaffolds were identified that activate Nanog expression in mouse fibroblasts transduced with a subset of reprogramming factors lacking Klf4. Application of one such molecule, kenpaullone, in lieu of Klf4 gave rise to iPS cells that are indistinguishable from murine embryonic stem cells. This experimental platform can be used to screen large chemical libraries in search of novel compounds to replace the reprogramming factors that induce pluripotency. Ultimately, such compounds may provide mechanistic insight into the reprogramming process.
特定转录因子的异位表达可将体细胞重编程为诱导多能干细胞(iPS细胞),但iPS细胞的应用因病毒递送系统的使用而受到阻碍。小分子提供了一种替代方法,可用负责重编程的化学信号线索取代病毒转导的转录因子。在本报告中,我们描述了一个小分子筛选平台,用于鉴定在功能上替代重编程因子Klf4的化合物。鉴定出了一系列小分子支架,它们可激活用缺乏Klf4的一部分重编程因子转导的小鼠成纤维细胞中的Nanog表达。应用一种这样的分子——肯帕罗宁代替Klf4,产生了与小鼠胚胎干细胞无法区分的iPS细胞。这个实验平台可用于筛选大型化学文库,以寻找新型化合物来替代诱导多能性的重编程因子。最终,此类化合物可能为深入了解重编程过程提供机制性见解。