临床级人多能干细胞用于细胞治疗：特征化策略。
Clinical-Grade Human Pluripotent Stem Cells for Cell Therapy: Characterization Strategy.
机构信息
Department of Experimental Biology, Faculty of Science, Masaryk University, Kamenice 5, 625 00 Brno, Czech Republic.
International Clinical Research Center, St. Anne's University Hospital Brno, Pekařská 53, 656 91 Brno, Czech Republic.
出版信息
Int J Mol Sci. 2020 Mar 31;21(7):2435. doi: 10.3390/ijms21072435.
Abstract
Human pluripotent stem cells have the potential to change the way in which human diseases are cured. Clinical-grade human embryonic stem cells and human induced pluripotent stem cells have to be created according to current good manufacturing practices and regulations. Quality and safety must be of the highest importance when humans' lives are at stake. With the rising number of clinical trials, there is a need for a consensus on hPSCs characterization. Here, we summarize mandatory and 'for information only' characterization methods with release criteria for the establishment of clinical-grade hPSC lines.
摘要
人类多能干细胞有可能改变人类疾病的治疗方式。根据现行的良好生产规范和法规，必须创建临床级别的人类胚胎干细胞和人类诱导多能干细胞。当涉及到人类的生命时，质量和安全必须是最重要的。随着临床试验数量的增加，需要就 hPSC 的特征达成共识。在这里，我们总结了强制性和“仅供参考”的特征分析方法以及建立临床级 hPSC 系的放行标准。
相似文献
1
Clinical-Grade Human Pluripotent Stem Cells for Cell Therapy: Characterization Strategy.临床级人多能干细胞用于细胞治疗：特征化策略。
Int J Mol Sci. 2020 Mar 31;21(7):2435. doi: 10.3390/ijms21072435.
2
Pluripotent Stem Cells in Clinical Setting-New Developments and Overview of Current Status.临床环境中的多能干细胞：新进展和现状概述。
Stem Cells. 2022 Sep 26;40(9):791-801. doi: 10.1093/stmcls/sxac040.
3
Recent Trends in Research with Human Pluripotent Stem Cells: Impact of Research and Use of Cell Lines in Experimental Research and Clinical Trials.人类多能干细胞研究的最新趋势：细胞系在实验研究和临床试验中的研究和应用的影响。
Stem Cell Reports. 2018 Aug 14;11(2):485-496. doi: 10.1016/j.stemcr.2018.06.012. Epub 2018 Jul 19.
4
Technological progress and challenges towards cGMP manufacturing of human pluripotent stem cells based therapeutic products for allogeneic and autologous cell therapies.基于人多能干细胞的治疗产品的 cGMP 制造的技术进展和挑战，用于同种异体和自体细胞治疗。
Biotechnol Adv. 2013 Dec;31(8):1600-23. doi: 10.1016/j.biotechadv.2013.08.009. Epub 2013 Aug 17.
5
The safety of human pluripotent stem cells in clinical treatment.人类多能干细胞在临床治疗中的安全性。
Ann Med. 2015;47(5):370-80. doi: 10.3109/07853890.2015.1051579. Epub 2015 Jul 6.
6
The Manufacture of Xeno- and Feeder-Free Clinical-Grade Human Embryonic Stem Cell Lines: First Step for Cell Therapy.无饲养层和动物源的临床级人胚胎干细胞系的制备：细胞治疗的第一步。
Int J Mol Sci. 2022 Oct 18;23(20):12500. doi: 10.3390/ijms232012500.
7
Derivation of Human Induced Pluripotent Stem Cell (iPSC) Lines and Mechanism of Pluripotency: Historical Perspective and Recent Advances.人类诱导多能干细胞（iPSC）系的建立和多能性的机制：历史视角和最新进展。
Stem Cell Rev Rep. 2017 Dec;13(6):757-773. doi: 10.1007/s12015-017-9766-9.
8
Haematopoietic developmental potential of human pluripotent stem cell lines.人类多能干细胞系的造血发育潜能。
Folia Biol (Praha). 2014;60 Suppl 1:90-4.
9
Human embryonic and induced pluripotent stem cells in clinical trials.临床试验中的人胚胎干细胞和诱导多能干细胞。
Br Med Bull. 2015;116:19-27. doi: 10.1093/bmb/ldv045. Epub 2015 Nov 17.
10
History and current status of clinical studies using human pluripotent stem cells.人类多能干细胞临床研究的历史和现状。
Stem Cell Reports. 2023 Aug 8;18(8):1592-1598. doi: 10.1016/j.stemcr.2023.03.005. Epub 2023 Apr 6.
引用本文的文献
1
Cell and tissue reprogramming: Unlocking a new era in medical drug discovery.细胞与组织重编程：开启药物研发的新时代。
Pharmacol Rev. 2025 Jun 26;77(5):100077. doi: 10.1016/j.pharmr.2025.100077.
2
Airway epithelial stem cell renewal and differentiation: overcoming challenging steps towards clinical-grade tissue engineering.气道上皮干细胞的更新与分化：克服迈向临床级组织工程的挑战性步骤。
Stem Cell Res Ther. 2025 Jul 6;16(1):351. doi: 10.1186/s13287-025-04478-0.
3
A comprehensive analysis of induced pluripotent stem cell (iPSC) production and applications.诱导多能干细胞（iPSC）生成与应用的综合分析。
Front Cell Dev Biol. 2025 May 8;13:1593207. doi: 10.3389/fcell.2025.1593207. eCollection 2025.
4
Methods for Pluripotent Stem Cell Characterization: A Narrative Review.多能干细胞鉴定方法：叙述性综述
Cureus. 2025 Jan 13;17(1):e77362. doi: 10.7759/cureus.77362. eCollection 2025 Jan.
5
Quality and Regulatory Requirements for the Manufacture of Master Cell Banks of Clinical Grade iPSCs: The EU and USA Perspectives.临床级诱导多能干细胞主细胞库生产的质量与监管要求：欧盟与美国视角
Stem Cell Rev Rep. 2025 Apr;21(3):645-679. doi: 10.1007/s12015-024-10838-9. Epub 2025 Jan 17.
6
Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice.人诱导多能干细胞衍生的具有放射状胶质细胞特征的神经干细胞在小鼠体内表现出长期安全性。
Nat Commun. 2024 Nov 1;15(1):9433. doi: 10.1038/s41467-024-53613-7.
7
Unlocking the therapeutic potential: odyssey of induced pluripotent stem cells in precision cell therapies.释放治疗潜力：诱导多能干细胞在精准细胞治疗中的探索历程
Int J Surg. 2024 Oct 1;110(10):6432-6455. doi: 10.1097/JS9.0000000000001892.
8
Challenges of CRISPR/Cas-Based Cell Therapy for Type 1 Diabetes: How Not to Engineer a "Trojan Horse".基于 CRISPR/Cas 的 1 型糖尿病细胞治疗的挑战：如何不制造“特洛伊木马”。
Int J Mol Sci. 2023 Dec 10;24(24):17320. doi: 10.3390/ijms242417320.
9
New characterization and safety evaluation of human limbal stem cells used in clinical application: fidelity of mitotic process and mitotic spindle morphologies.临床应用中人角膜缘干细胞的新特征和安全性评估：有丝分裂过程和有丝分裂纺锤体形态的保真度。
Stem Cell Res Ther. 2023 Dec 13;14(1):368. doi: 10.1186/s13287-023-03586-z.
10
iPSC-based approach for human hair follicle regeneration.基于诱导多能干细胞的人类毛囊再生方法。
Front Cell Dev Biol. 2023 May 30;11:1149050. doi: 10.3389/fcell.2023.1149050. eCollection 2023.
本文引用的文献
1
Quantification of the morphological characteristics of hESC colonies.人胚胎干细胞集落形态特征的定量分析。
Sci Rep. 2019 Nov 26;9(1):17569. doi: 10.1038/s41598-019-53719-9.
2
Enhanced wound healing promotion by immune response-free monkey autologous iPSCs and exosomes vs. their allogeneic counterparts.免疫反应-free 猴自体 iPSCs 和外泌体对创面愈合的促进作用优于其同种异体对应物。
EBioMedicine. 2019 Apr;42:443-457. doi: 10.1016/j.ebiom.2019.03.011. Epub 2019 Mar 26.
3
GMP-compatible manufacturing of three iPS cell lines from human peripheral blood.符合药品生产质量管理规范（GMP）的源自人外周血的三种诱导多能干细胞系的制造。
Stem Cell Res. 2019 Mar;35:101394. doi: 10.1016/j.scr.2019.101394. Epub 2019 Feb 11.
4
Direct application of induced pluripotent stem cells is feasible and can be safe.直接应用诱导多能干细胞是可行的，也是安全的。
Theranostics. 2019 Jan 1;9(1):290-310. doi: 10.7150/thno.28671. eCollection 2019.
5
Next generation sequencing-based gene panel tests for the management of solid tumors.基于下一代测序的基因面板检测在实体瘤管理中的应用。
Cancer Sci. 2019 Jan;110(1):6-15. doi: 10.1111/cas.13837. Epub 2018 Nov 27.
6
Quality control guidelines for clinical-grade human induced pluripotent stem cell lines.临床级人诱导多能干细胞系的质量控制指南。
Regen Med. 2018 Oct;13(7):859-866. doi: 10.2217/rme-2018-0095. Epub 2018 Sep 12.
7
Human-Induced Pluripotent Stem Cells Manufactured Using a Current Good Manufacturing Practice-Compliant Process Differentiate Into Clinically Relevant Cells From Three Germ Layers.采用符合现行药品生产质量管理规范的工艺制造的人诱导多能干细胞可分化为来自三个胚层的临床相关细胞。
Front Med (Lausanne). 2018 Mar 15;5:69. doi: 10.3389/fmed.2018.00069. eCollection 2018.
8
Concise Review: Assessing the Genome Integrity of Human Induced Pluripotent Stem Cells: What Quality Control Metrics?简明综述：评估人类诱导多能干细胞的基因组完整性：有哪些质量控制指标？
Stem Cells. 2018 Jun;36(6):814-821. doi: 10.1002/stem.2797. Epub 2018 Feb 18.
9
Monitoring cell line identity in collections of human induced pluripotent stem cells.监测人类诱导多能干细胞库中的细胞系身份。
Stem Cell Res. 2018 Apr;28:66-70. doi: 10.1016/j.scr.2018.01.030. Epub 2018 Jan 31.
10
Lessons for reviewing clinical trials using induced pluripotent stem cells: examining the case of a first-in-human trial for age-related macular degeneration.使用诱导多能干细胞回顾临床试验的经验教训：以年龄相关性黄斑变性的首次人体试验为例进行审视。
Regen Med. 2018 Mar;13(2):123-128. doi: 10.2217/rme-2017-0130. Epub 2017 Dec 6.
Zotero 插件