Safety Assessment of Pluripotent Stem Cell Therapy Using Nanotechnology

描述(申请人提供)：人类多能干细胞是再生医学的有前途的和潜在的无限细胞来源；然而，肿瘤的形成是多能干细胞细胞治疗的主要问题。干细胞衍生品中残留的未分化干细胞，包括心肌细胞分化培养，在移植后可能会增殖并形成畸胎瘤，这是安全细胞治疗的主要障碍。仔细评估分化培养和开发去除未分化干细胞的策略将有助于鉴定用于治疗的最终干细胞产品。在这项研究中，我们建议开发敏感的、基于纳米技术的分析来检测残留的未分化细胞，表征不同阶段的心肌分化培养细胞形成畸胎瘤的倾向，并评估选择性去除残留的未分化细胞的策略。这项研究建立的技术将对利用人类多能干细胞进行新型细胞疗法的开发具有价值。

DESCRIPTION (provided by applicant): Human pluripotent stem cells are promising and potentially unlimited cell source for regenerative medicine; however, tumor formation is of major concern for cell therapy with pluripotent stem cells. Residual undifferentiated stem cells in preparations of stem cell derivatives, including cardiomyocyte differentiation cultures, have potential to proliferate and form teratoma upon transplantation, posing a major hurdle for safe cell therapy. Careful assessment of differentiation cultures and development of strategies to remove undifferentiated stem cells will facilitate qualification of final stem cell products for therapeutic use. In this study, we propose to develop sensitive, nanotechnology-based assays to detect residual undifferentiated cells, characterize cardiomyocyte differentiation cultures at various stages for their propensities to form teratomas, and evaluate strategies to selectively remove residual undifferentiated cells. Technology established in this study will be valuable for the development of novel cell therapy using human pluripotent stem cells.