Department of Clinical Medicine (K1), University of Bergen, Bergen, Norway.
Int J Biochem Cell Biol. 2024 May;170:106556. doi: 10.1016/j.biocel.2024.106556. Epub 2024 Feb 27.
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.
线粒体疾病由于线粒体功能障碍的复杂性和多面性而难以治疗。脑类器官是源自人类多能干细胞的三维（3D）结构，旨在模拟类脑发育和功能。脑类器官作为模拟人类疾病、大脑发育和药物筛选的强大工具，近年来受到了广泛关注。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。脑类器官为个性化医疗提供了可能，因为它们可以从患者特异性细胞中获得，从而可以测试针对特定基因突变的药物。在本文中，我们强调了脑类器官如何为筛选线粒体疾病化合物提供有前途的途径，并讨论了与它们的使用相关的挑战和局限性。我们希望这篇综述将为线粒体筛选研究中脑类器官的进一步进展提供新的见解。
