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

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机构： [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.

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关键词： acute brain injury bioengineering brain organoids brain tissue transplantation cerebral organoids drug screening induced pluripotent stem cell neurodegenerative diseases neurodevelopmental disorders

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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.Copyright © 2025 Neural Regeneration Research.

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大类 | 2 区医学

小类 | 2 区细胞生物学 2 区神经科学

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第一作者机构： [1]Department of Anesthesiology, The Third Affiliated Hospital of Zunyi Medical University (The First People's Hospital of Zunyi), Zunyi, Guizhou Province, China.

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