Human brain organoids: an innovative model for neurological disorder research and therapy.

IF 4 3区医学 Q2 NEUROSCIENCES

Frontiers in Cellular Neuroscience Pub Date : 2025-08-26 eCollection Date: 2025-01-01 DOI:10.3389/fncel.2025.1658074

Hancheng Li, Junxiao Zhu, Jieyu Li, Yangkai Wu, Chaohua Luo, Yuting Huang, Jieru Wu, Wenhua Liu, Hongwu Wang, Zhixian Mo

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Abstract

The emergence of human brain organoids (hBOs) has transformed how we study brain development, disease mechanisms, and therapy discovery. These 3D in vitro 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.

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人脑类器官：神经系统疾病研究和治疗的创新模型。

人脑类器官（hBOs）的出现改变了我们研究大脑发育、疾病机制和治疗发现的方式。这些3D体外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，为研究神经发育和神经疾病提供了一个超越传统2D培养和动物模型的开创性平台。为了进一步探索hBOs技术的潜力，我们回顾了目前的文献，特别关注其在诊断和治疗主要神经系统疾病（如阿尔茨海默病、帕金森病和其他相关神经系统疾病）方面的应用。利用患者来源的诱导多能干细胞与尖端的基因编辑技术相结合，hBOs能够进行高精度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的优点和目前的局限性。尽管存在这些挑战，hBOs仍然是靶向神经治疗发展的变革性平台。总的来说，这篇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。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Frontiers in Cellular Neuroscience NEUROSCIENCES-

CiteScore

7.90

自引率

3.80%

发文量

627

审稿时长

6-12 weeks

期刊介绍： Frontiers in Cellular Neuroscience is a leading journal in its field, publishing rigorously peer-reviewed research that advances our understanding of the cellular mechanisms underlying cell function in the nervous system across all species. Specialty Chief Editors Egidio D‘Angelo at the University of Pavia and Christian Hansel at the University of Chicago are supported by an outstanding Editorial Board of international researchers. This multidisciplinary open-access journal is at the forefront of disseminating and communicating scientific knowledge and impactful discoveries to researchers, academics, clinicians and the public worldwide.