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The Application of Brain Organoid for Drug Discovery in Mitochondrial Diseases
The International Journal of Biochemistry & Cell Biology ( IF 4 ) Pub Date : 2024-02-27 , DOI: 10.1016/j.biocel.2024.106556
Kristina Xiao Liang

Mitochondrial diseases are difficult to treat due to the complexity and multifaceted nature of mitochondrial dysfunction. Brain organoids are three-dimensional (3D) structures derived from human pluripotent stem cells designed to mimic brain-like development and function. Brain organoids have received a lot of attention in recent years as powerful tools for modeling human diseases, brain development, and drug screening. Screening compounds for mitochondrial diseases using brain organoids could provide a more physiologically relevant platform for drug discovery. Brain organoids offer the possibility of personalized medicine because they can be derived from patient-specific cells, allowing testing of drugs tailored to specific genetic mutations. In this article, we highlight how brain organoids offer a promising avenue for screening compounds for mitochondrial diseases and address the challenges and limitations associated with their use. We hope this review will provide new insights into the further progress of brain organoids for mitochondrial screening studies.

中文翻译:

脑类器官在线粒体疾病药物发现中的应用

由于线粒体功能障碍的复杂性和多方面性,线粒体疾病难以治疗。脑类器官是源自人类多能干细胞的三维 (3D) 结构,旨在模拟类脑的发育和功能。近年来,脑类器官作为人类疾病建模、大脑发育和药物筛选的强大工具受到了广泛关注。使用脑类器官筛选线粒体疾病化合物可以为药物发现提供更具生理相关性的平台。脑类器官提供了个性化医疗的可能性,因为它们可以源自患者特异性细胞,从而可以测试针对特定基因突变的药物。在本文中,我们重点介绍了脑类器官如何为筛选线粒体疾病化合物提供有前景的途径,并解决与其使用相关的挑战和限制。我们希望这篇综述能为线粒体筛选研究的脑类器官的进一步进展提供新的见解。
更新日期:2024-02-27
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