中国神经再生研究(英文版) ›› 2026, Vol. 21 ›› Issue (10): 4734-4744.doi: 10.4103/NRR.NRR-D-25-00924

• 综述:退行性病与再生 • 上一篇    下一篇

类器官:神经退行性疾病研究的关键进展、优化与技术迭代

  

  • 出版日期:2026-10-15 发布日期:2026-06-12
  • 基金资助:
     国家重点研发计划(2021YFA1101802)、国家自然科学基金(82371260、U23A20429)

Organoids: Key advances, optimization, and technological iterations in their application to neurodegenerative diseases.

Jiangyu Zhao, Jing Wang*, Xing Guo*   

  1. Department of Neurobiology, School of Basic Medical Sciences, Nanjing Medical University, Nanjing, Jiangsu Province, China
  • Online:2026-10-15 Published:2026-06-12
  • Contact: Xing Guo, PhD, guox@njmu.edu.cn; Jing Wang, PhD, wangjing@njmu.edu.cn.
  • Supported by:
    This work was jointly supported by the National Key Research and Development Program of China, No. 2021YFA1101802, the National Natural Science Foundation of China, No. 82371260, U23A20429 (all to XG).

摘要:

类器官技术作为一种创新手段,已被证明在疾病建模、靶点筛选及治疗策略制定中具有巨大潜力。然而,传统类器官在研究中仍存在3大主要局限:特定细胞类型缺失、血脑屏障结构缺失以及实验结果的可重复性不足。近年来,研究者通过引入先进培养技术、微流控系统、生物打印、类器官移植及组装体构建等创新手段,逐步克服了这些局限性,推动了类器官技术在神经退行性疾病研究中的广泛应用。文章的目的是系统梳理类器官在神经退行性疾病研究中的技术创新。通过总结经典类器官构建方案及其局限性,强调了类器官技术在神经发育障碍研究中的全流程应用价值。针对阿尔茨海默病、帕金森病、亨廷顿病、肌萎缩侧索硬化症及额颞叶痴呆等5类疾病的研究表明,类器官技术显著提升了实验的可及性,并缩短了疾病建模、靶点发现及治疗开发的周期。通过定制化设备与基因编辑技术,类器官能够精准匹配特定需求,为构建病理生理学相关的疾病模型提供了重要工具,从而推动了对神经发育障碍的深入认知。尽管类器官技术在疾病研究中已展现出显著优势,但其在治疗神经退行性疾病方面的潜力尚未充分挖掘,这可能成为未来研究的重要方向。


https://orcid.org/0000-0002-0216-0310 (Xing Guo); https://orcid.org/0000-0002-3075-2874 (Jing Wang)

关键词: 阿尔茨海默病, 肌萎缩侧索硬化症, 生物打印, 额颞叶痴呆, 亨廷顿病, 微流控技术, 神经退行性疾病, 类器官, 帕金森病, 移植

Abstract: Organoid technology, as an innovative approach, has shown great potential in disease modeling, target screening, and the development of treatment strategies. However, traditional organoids still have three major limitations in research: the absence of specific cell types, the lack of blood–brain barrier structure, and insufficient reproducibility of experimental results. In recent years, researchers have gradually overcome these limitations by introducing innovative techniques such as advanced culture methods, microfluidic systems, bioprinting, organoid transplantation, and assembloid construction. This progress has facilitated the widespread application of organoids in the study of neurodegenerative diseases. This paper aims to systematically review the technological innovations of organoids in the study of neurodegenerative diseases. By summarizing classical organoid construction strategies and their limitations, it emphasizes the value of organoids in comprehensive applications within neurodegenerative disease research. In this review, we focus on five specific neurodegenerative diseases: Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis, and frontotemporal dementia. Research in these diseases demonstrates that organoids improve experimental accessibility and reduce development cycles in disease modeling, target discovery, and therapeutic strategy formation. Using customized equipment and gene editing techniques, these organoids can be tailored to specific needs, providing pathophysiologically relevant disease models and enhancing our understanding of neurodegenerative diseases. Although organoid technology has demonstrated significant advantages in disease research, its potential for treating neurodegenerative diseases has not yet been fully explored, which may become an important direction for future research.

Key words: Alzheimer disease, amyotrophic lateral sclerosis, bioprinting, frontotemporal dementia, Huntington’s disease, microfluidics, neurodegenerative diseases, organoids, Parkinson’s disease, transplantation