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

利用基因治疗生物技术和胚胎干细胞开发针对先天性疾病的新型再生医学

• 批准号: 14570737
• 负责人: KOSAI Ken-ichiro
• 金额: $ 2.5万
• 依托单位: Kurume University (2003)Gifu University (2002)
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Scientific Research (C)
• 财政年份: 2002
• 资助国家: 日本
• 起止时间: 2002 至 2003
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/en/grant/KAKENHI-PROJECT-14570737/
• 关键词: ES cell; Gene transfer; Vector; Congenital disease; Regenerative medicine; Adenoviral vector; Gene expression; Cardiomyocyte

<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.

再生医学和基因疗法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)对照诱导胚胎干细胞(ES)来源的靶细胞；2)对ES来源的靶细胞进行一定的纯化。2.我们开发了一种新的方法，可以有效地诱导成纤维细胞生长因子-2和骨形态发生蛋白-2诱导ES细胞向心肌分化。3.为了进一步将这项技术用于未来的临床应用，我们正在进行使用人类ES细胞的研究(我们的方案已正式获得批准)。&lt；Future plan&gt；1.我们将使用纯化的ES来源的靶细胞克隆重要的基因。2.我们将评估我们的战略的治疗潜力。3.我们将继续使用人ES细胞和小鼠ES细胞进行这项研究。

项目成果

Terazaki Y., et al.Kosai K: "An optimal therapeutic expression level is crucial for suicide gene therapy for hepatic metastatic cancer in mice."Hepatology. 37. 155-163 (2003)

Terazaki Y. 等人 Kosai K：“最佳治疗表达水平对于小鼠肝转移癌的自杀基因治疗至关重要。” 肝病学。

Himeno W. et al.: "Increased angiogenic growth factor in cyanotic congenital heart disease."Pediatr Cardiol. 24(2). 127-132 (2003)

Himeno W. 等人：“紫绀型先天性心脏病中血管生成生长因子增加。”Pediatr Cardiol。

Iida SI, Hirota T, Morisaki T, Marumoto T, Hara T, Kuninaka S, Kosai K, et al.: "suppressor WARTS ensures genomic integrity by regulating both mitotic progression and G(1) tetraploidy checkpoint function"Oncogene.. (in press). (2004)

Iida SI、Hirota T、Morisaki T、Marumoto T、Hara T、Kuninaka S、Kosai K 等人：“抑制子 WARTS 通过调节有丝分裂进程和 G(1) 四倍体检查点功能来确保基因组完整性”Oncogene..（in

Himeno W, Kosai K, et al.: "Increased angiogenic growth factor in cyanotic congenital heart disease."Pediatr Cardiol. 24(2). 127-132 (2003)

Himeno W、Kosai K 等人：“紫绀型先天性心脏病中血管生成生长因子增加。”Pediatr Cardiol。

Hisaka Y, Kosai K, et al.: "Powerful and controllable angiogenesis by using gene-modified cells expressing human hepatocyte growth factor and thymidine kinase."J Am Coll Cardiol.. 43. 1915-1922 (2004)

Hisaka Y、Kosai K 等人：“通过使用表达人肝细胞生长因子和胸苷激酶的基因修饰细胞实现强大且可控的血管生成。”J Am Coll Cardiol.. 43. 1915-1922 (2004)