Safety Assessment of Pluripotent Stem Cell Therapy Using Nanotechnology

使用纳米技术的多能干细胞疗法的安全性评估

基本信息

批准号：
8752641
负责人：
Chunhui Xu
金额：
$ 23.4万
依托单位：
EMORY UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2014
资助国家：
美国
起止时间：
2014-08-15 至 2016-04-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8752641
关键词：
Biological Biological Assay Cardiac Cardiac Myocytes Cell Differentiation process Cell Therapy Cells Clinical Complex Development Diagnostic Disease Environment Evaluation Fingerprint Flow Cytometry Gene Expression Genes Heart failure Human In Vitro Molecular Molecular Profiling Nanotechnology Outcome Pathway interactions Pattern Pluripotent Stem Cells Population Preparation Proliferating Raman Spectrum Analysis Regenerative Medicine Residual state Risk Safety Source Specificity Staging Stem cells Surface Technology Teratoma Therapeutic Uses Translations Transplantation Tumorigenicity Undifferentiated base cell preparation cell type clinical application design human embryonic stem cell in vivo induced pluripotent stem cell meetings nanoparticle novel novel strategies prevent public health relevance single molecule stem cell therapy tool tumor

项目摘要

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.

描述（由申请人提供）：人类多能干细胞对于再生医学来说是有前景且可能无限的细胞来源；然而，肿瘤形成是多能干细胞细胞治疗的主要关注点。干细胞衍生物制剂（包括心肌细胞分化培养物）中残留的未分化干细胞在移植后有可能增殖并形成畸胎瘤，这对安全细胞治疗构成了主要障碍。仔细评估分化培养物并制定去除未分化干细胞的策略将有助于最终干细胞产品用于治疗用途的资格。在这项研究中，我们建议开发灵敏的、基于纳米技术的检测方法来检测残留的未分化细胞，表征不同阶段的心肌细胞分化培养物形成畸胎瘤的倾向，并评估选择性去除残留的未分化细胞的策略。这项研究中建立的技术对于开发利用人类多能干细胞的新型细胞疗法具有重要价值。