Li Hancheng, Zhu Junxiao, Li Jieyu, Wu Yangkai, Luo Chaohua, Huang Yuting, Wu Jieru, Liu Wenhua, Wang Hongwu, Mo Zhixian
Department of Pharmaceutical Engineering, School of Food and Pharmaceutical Engineering, Zhaoqing University, Zhaoqing, China.
Department of Pharmacology of Chinese Medicine, School of Traditional Chinese Medicine, Southern Medical University, Guangzhou, China.
Front Cell Neurosci. 2025 Aug 26;19:1658074. doi: 10.3389/fncel.2025.1658074. eCollection 2025.
The emergence of human brain organoids (hBOs) has transformed how we study brain development, disease mechanisms, and therapy discovery. These 3D neural models closely mimic the cellular diversity, spatial structure, and functional connectivity of the human brain, providing a groundbreaking platform that outperforms traditional 2D cultures and animal models in studying neurodevelopment and neurological disorders. To further explore the potential of hBOs technology, we review current literature focusing particularly on its applications for diagnosing and treating major neurological diseases such as Alzheimer's disease, Parkinson's disease, and other related neurological disorders. Using patient-derived induced pluripotent stem cells combined with cutting-edge gene-editing technologies, hBOs enable highly precise mechanistic studies and scalable drug screening. Moreover, we further discuss the advantages and current limitations of hBOs. Despite these challenges, hBOs remain a transformative platform for the development of targeted neurotherapeutics. Collectively, this review offers a solid foundation for advancing neuroscience research and fostering innovative treatment strategies for neurological disorders.
人脑类器官(hBOs)的出现改变了我们研究大脑发育、疾病机制和治疗方法的方式。这些三维神经模型紧密模拟了人类大脑的细胞多样性、空间结构和功能连接性,提供了一个开创性的平台,在研究神经发育和神经系统疾病方面优于传统的二维培养和动物模型。为了进一步探索hBOs技术的潜力,我们回顾了当前的文献,特别关注其在诊断和治疗阿尔茨海默病、帕金森病等主要神经系统疾病以及其他相关神经系统疾病中的应用。利用患者来源的诱导多能干细胞结合前沿基因编辑技术,hBOs能够进行高度精确的机制研究和可扩展的药物筛选。此外,我们进一步讨论了hBOs的优势和当前的局限性。尽管存在这些挑战,hBOs仍然是开发靶向神经治疗药物的变革性平台。总体而言,本综述为推进神经科学研究和促进神经系统疾病的创新治疗策略提供了坚实的基础。