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利用人多能干细胞衍生的心肌细胞加强心脏治疗产品线。

Strengthening cardiac therapy pipelines using human pluripotent stem cell-derived cardiomyocytes.

作者信息

Raniga Kavita, Nasir Aishah, Vo Nguyen T N, Vaidyanathan Ravi, Dickerson Sarah, Hilcove Simon, Mosqueira Diogo, Mirams Gary R, Clements Peter, Hicks Ryan, Pointon Amy, Stebbeds Will, Francis Jo, Denning Chris

机构信息

The Biodiscovery Institute, University of Nottingham, Nottingham NG7 2RD, UK; Pathology, Non-Clinical Safety, GlaxoSmithKline R&D, Stevenage SG1 2NY, UK.

The Biodiscovery Institute, University of Nottingham, Nottingham NG7 2RD, UK.

出版信息

Cell Stem Cell. 2024 Mar 7;31(3):292-311. doi: 10.1016/j.stem.2024.01.007. Epub 2024 Feb 15.

DOI:10.1016/j.stem.2024.01.007
PMID:38366587
Abstract

Advances in hiPSC isolation and reprogramming and hPSC-CM differentiation have prompted their therapeutic application and utilization for evaluating potential cardiovascular safety liabilities. In this perspective, we showcase key efforts toward the large-scale production of hiPSC-CMs, implementation of hiPSC-CMs in industry settings, and recent clinical applications of this technology. The key observations are a need for traceable gender and ethnically diverse hiPSC lines, approaches to reduce cost of scale-up, accessible clinical trial datasets, and transparent guidelines surrounding the safety and efficacy of hiPSC-based therapies.

摘要

人诱导多能干细胞(hiPSC)分离、重编程以及人多能干细胞来源的心肌细胞(hPSC-CM)分化技术的进展推动了它们在治疗方面的应用,以及用于评估潜在心血管安全风险。从这个角度来看,我们展示了在大规模生产hPSC-CM、在工业环境中应用hPSC-CM以及该技术近期临床应用方面所做的关键努力。主要发现包括需要可追溯的不同性别和种族的hiPSC系、降低扩大规模成本的方法、可获取的临床试验数据集,以及围绕基于hiPSC疗法的安全性和有效性的透明指南。

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