Development of novel regenerative medicine for congenital diseases using gene therapy biotechnology and embryonic stem cell

<Aim>再生医学和基因治疗可能是许多先天性疾病最有前途的治疗方法。在这方面，开发一种新的生物技术和治疗策略是至关重要的。本研究开发了以下技术：1）控制从胚胎干（ES）细胞诱导靶细胞，2）ES衍生的靶细胞的一定纯化。<Results>1.一、我们开发了一种新的生物技术，有助于确定纯化ES衍生的靶细胞。2.我们开发了一种新的方法，有效地诱导心肌分化的ES细胞使用FGF-2和BMP-2。3.为了进一步利用这项技术用于未来的临床应用，我们正在使用人类ES细胞进行研究（我们的方案已正式批准）。<Future plan>1.一、我们将使用纯化的ES衍生的靶细胞克隆重要基因。2.我们将评估我们策略的治疗潜力。3.我们将继续使用人类ES细胞以及小鼠ES细胞进行这项研究。

<Aim> Regenerative medicine and gene therapy may be the most promising therapeutics for many of congenital diseases. In this respect, a development of a novel biotechnology and therapeutic strategy is crucially necessary. The present study develops the following technology; 1) control to induce a target cell from embryonic stem (ES) cell, 2) certain purification of ES-derived target cells.<Results> 1. We developed a novel biotechnology that facilitated to certainly purify ES-derived target cells. 2. We developed a novel method that efficiently induced cardiomyogenic differentiation of ES cells using FGF-2 and BMP-2. 3. To further utilize this technology for future clinical application, we are doing research using human ES cells (our protocol was officially approved).<Future plan> 1. We will clone important genes using purified ES-derived target cells. 2. We will assess the therapeutic potential of our strategy. 3. We will continue this research using human ES cells as well as mouse ES cell.