Varga Eszter, Nemes Csilla, Bock István, Táncos Zsuzsanna, Berzsenyi Sára, Lévay György, Román Viktor, Kobolák Julianna, Dinnyés András
BioTalentum Ltd., Gödöllő, Hungary.
Pharmacology and Drug Safety Research, Gedeon Richter Plc., Budapest, Hungary.
Stem Cell Res. 2017 May;21:19-22. doi: 10.1016/j.scr.2017.03.013. Epub 2017 Mar 18.
Peripheral blood mononuclear cells (PBMCs) were collected from a clinically characterized patient with autism spectrum disorder (ASD). The PMBCs were reprogrammed with the human OSKM transcription factors using the Sendai-virus delivery system. The pluripotency of transgene-free iPSCs was verified by immunocytochemistry for pluripotency markers and by spontaneous in vitro differentiation towards the 3 germ layers. Furthermore, the iPSC line showed normal karyotype. Our model might offer a good platform to study the pathomechanism of ASD, also for drug testing, early biomarker discovery and gene therapy studies.
从一名具有临床特征的自闭症谱系障碍（ASD）患者中采集外周血单个核细胞（PBMC）。使用仙台病毒递送系统，用人OSKM转录因子对PBMC进行重编程。通过对多能性标志物进行免疫细胞化学检测以及对3个胚层进行自发体外分化，验证了无转基因诱导多能干细胞（iPSC）的多能性。此外，该iPSC系显示出正常的核型。我们的模型可能为研究ASD的发病机制提供一个良好的平台，也可用于药物测试、早期生物标志物发现和基因治疗研究。
