中国神经再生研究(英文版) ›› 2026, Vol. 21 ›› Issue (7): 2580-2600.doi: 10.4103/NRR.NRR-D-24-01490

• 综述:神经损伤修复保护与再生 • 上一篇    下一篇

脑类器官在中枢神经系统疾病中的进展与应用:从实验室到临床的桥梁

  

  • 出版日期:2026-07-15 发布日期:2025-10-17

Advances and applications of brain organoids in central nervous system disorders: Bridging the gap from laboratory to clinic

Changle Fang1, #, Qiulin Wang1, #, Qiuxia Xiao1 , Xiaoxing Cai2 , Ruolan Du3 , Lulu Xue4 , Xiaohe Tian1, *, Liulin Xiong1, *   

  1. 1 Department of Anesthesiology, The Third Affiliated Hospital of Zunyi Medical University (The First People’s Hospital of Zunyi), Zunyi, Guizhou Province, China;  2 Institute of Neurosciences, Kunming Medical University, Kunming, Yunnan Province, China;  3 Institute of Neurological Disease, National-Local Joint Engineering Research Center of Translational Medicine, West China Hospital of Sichuan University, Chengdu, Sichuan Province, China;  4 State Key Laboratory of Biotherapy, Sichuan University, Chengdu, Sichuan Province, China
  • Online:2026-07-15 Published:2025-10-17
  • Contact: Liulin Xiong, MD, PhD, liulin.xiong@mymail.unisa.edu.au; Xiaohe Tian, PhD, xiaohe.t@wchscu.cn.
  • Supported by:
    This work was supported by Guizhou Provincial Higher Education Science and Technological Innovation Team, No. [2023]072 (to LX); Graduate Education and Teaching Innovation Program of Zunyi Medical University, No. ZYK262 (to QW); and the Guizhou Graduate Research Fund, No. 2024YJSKYJJ333 (to QW).

摘要:

由于人脑的不可接近性和复杂性,中枢神经系统疾病的病理生理学研究一直是一个难题。脑器质体是由多能干细胞自组装而成的三维聚合体,其细胞类型和结构与胚胎人脑相似。脑器质体显示出研究中枢神经系统疾病所强调的非典型细胞、分子和遗传特征的潜力。此外,脑器官组织还为药物筛选提供了一个平台,并可作为脑损伤移植治疗的潜在来源。然而,脑组织器官的广泛应用受到一些限制因素的阻碍,如缺乏高保真细胞类型、成熟度不够以及异质性较大。这些局限性削弱了脑器官组织在具体应用中的可靠性。此综述总结了脑类器官在中枢神经系统疾病中的进展与应用进展,讨论了近年来的技术和方法创新,回顾了在药物筛选、移植治疗和疾病建模方面的应用以及临床研究的进展。此外,还强调了目前类脑器官研究的局限性,并探讨了该技术的发展潜力,以提高其适用性。

https://orcid.org/0000-0002-1623-5969 (Liulin Xiong); https://orcid.org/0000-0002-2294-3945 (Xiaohe Tian)

关键词: 性脑损伤, 生物工程, 脑类器官, 脑组织移植, 药物筛选, 诱导多能干细胞, 神经退行性疾病, 神经发育障碍

Abstract: Investigating the mechanisms underlying central nervous system disorders is a major scientific issue in the 21st century. However, the inaccessibility and complexity of the human brain have always represented a challenge in understanding the pathophysiology of the central nervous system. Brain organoids are self-assembled three-dimensional aggregates derived from pluripotent stem cells with cell types and structures similar to the embryonic human brain, giving them potential for investigating the atypical cellular, molecular, and genetic characteristics characteristic of central nervous system disorders. Brain organoids also provide a platform for drug screening and serve as a potential source for transplantation therapy for brain injuries. However, the broad application of brain organoids is hampered by several limitations, such as the lack of high-fidelity cell types, insufficient maturation, and considerable heterogeneity, undermining their reliability in specific applications. This review summarizes brain organoid evolution, discusses recent technological and methodological innovations, and reviews their applications in drug screening, transplantation therapy, and disease modeling, as well as clinical research progress. Additionally, we emphasize the limitations of current brain organoid research and explore the potential for advancing the technology to enhance its applicability.

Key words: acute brain injury, bioengineering, brain organoids, brain tissue transplantation, cerebral organoids, drug screening, induced pluripotent stem cell, neurodegenerative diseases, neurodevelopmental disorders