Yurdakul-Mesutoglu Pinar, Akin Hasan Yalim, Gunaydin Zeynep
Department of Medical Microbiology, Istinye University Faculty of Medicine, Istanbul, Turkey.
Ankara University Faculty of Medicine, Cord Blood Bank, Ankara, Turkey.
Methods Mol Biol. 2025;2960:57-84. doi: 10.1007/7651_2025_610.
Umbilical cord blood (CB)-derived hematopoietic stem and progenitor cells (HSPCs) hold immense potential for regenerative medicine, particularly in hematologic malignancies and immune disorders. CB offers several advantages, including easy collection and reduced risk of graft-versus-host disease compared to other sources, like bone marrow. However, the clinical application of CB is often limited due to the relatively small number of HSPCs present in CB grafts, which can be insufficient for adult patients. This limitation has prompted researchers to explore various methods to expand HSPCs ex vivo. As research continues to refine expansion techniques, the future of CB HSPC therapy appears increasingly promising, offering new hope for patients requiring stem cell transplantation. Approaches to HSPC expansion include the use of cytokines, small molecules, epigenetic modulators, and advanced culture systems that mimic the bone marrow niche as well as emerging techniques such as gene editing. Of the key CB HSPC expansion methodologies, the use of epigenetic modifiers is among the most promising strategies for inducing proliferation while maintaining the stemness of CB HSPC. This section summarizes key methodologies for CB HSPC expansion and their transformative impact on clinical practice while providing a validated protocol for ex vivo expansion of CB-derived HSPCs using valproic acid and/or nicotinamide.
脐带血(CB)来源的造血干细胞和祖细胞(HSPCs)在再生医学中具有巨大潜力,尤其是在血液系统恶性肿瘤和免疫疾病方面。与骨髓等其他来源相比,脐带血具有诸多优势,包括采集简便以及移植物抗宿主病风险降低。然而,由于脐带血移植物中存在的造血干细胞数量相对较少,这对于成年患者可能不足,因此脐带血的临床应用常常受到限制。这一限制促使研究人员探索各种体外扩增造血干细胞的方法。随着研究不断完善扩增技术,脐带血造血干细胞治疗的前景似乎越来越广阔,为需要干细胞移植的患者带来了新的希望。造血干细胞扩增方法包括使用细胞因子、小分子、表观遗传调节剂以及模拟骨髓微环境的先进培养系统,还有诸如基因编辑等新兴技术。在关键的脐带血造血干细胞扩增方法中,使用表观遗传修饰剂是诱导增殖同时维持脐带血造血干细胞干性的最具前景的策略之一。本节总结了脐带血造血干细胞扩增的关键方法及其对临床实践的变革性影响,同时提供了使用丙戊酸和/或烟酰胺体外扩增脐带血来源造血干细胞的有效方案。