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疾病建模与再生医学中的类器官

Organoids in disease modeling and regenerative medicine.

作者信息

Morizane Ryuji, Lamers Mart M

机构信息

Nephrology Division, Massachusetts General Hospital, Boston, Massachusetts, 02114, USA.

Department of Medicine, Harvard Medical School, Boston, Massachusetts, 02115, USA.

出版信息

Cell Mol Life Sci. 2025 Apr 21;82(1):169. doi: 10.1007/s00018-025-05692-y.

Abstract

Organoid technology has the potential to revolutionize biomedical research by providing more physiologically relevant models for studying human development, disease mechanisms, and therapeutic development. Derived from stem cells, organoids self-organize into three-dimensional tissues that replicate the structures and functions of their in vivo counterparts. Their ability to mimic organ-specific microstructures offers new tools for investigating organogenesis, modeling genetic disorders, and screening potential therapeutics using human cells. Additionally, organoids hold promise for regenerative medicine as potential transplantable tissues for repairing or replacing damaged organs. However, challenges such as batch variability, standardization, vascularization, long-term viability, and lack of immune cells remain, hindering their clinical translation and use in disease studies. Recent efforts have focused on improving reproducibility, incorporating bioengineering techniques for enhanced maturation, and optimizing differentiation methods. This collection highlights recent advances in the respiratory, renal, and retinal organoid systems. From refining cryopreservation methods to using organoid models for virus neutralization and inflammatory studies, these contributions emphasize the potential of organoids in translational research and regenerative medicine.

摘要

类器官技术有潜力通过提供更具生理相关性的模型来彻底改变生物医学研究,这些模型可用于研究人类发育、疾病机制和治疗开发。类器官由干细胞衍生而来,能自我组织形成三维组织,复制其体内对应组织的结构和功能。它们模拟器官特异性微观结构的能力为研究器官发生、模拟遗传疾病以及使用人类细胞筛选潜在治疗方法提供了新工具。此外,类器官作为用于修复或替换受损器官的潜在可移植组织,在再生医学方面具有广阔前景。然而,诸如批次变异性、标准化、血管化、长期生存能力以及缺乏免疫细胞等挑战依然存在,阻碍了它们在临床转化以及疾病研究中的应用。近期的努力集中在提高可重复性、纳入生物工程技术以促进成熟以及优化分化方法。本合集重点介绍了呼吸、肾脏和视网膜类器官系统的最新进展。从改进冷冻保存方法到使用类器官模型进行病毒中和及炎症研究,这些成果强调了类器官在转化研究和再生医学中的潜力。

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