The Application of Brain Organoid for Drug Discovery in Mitochondrial Diseases

Int J Biochem Cell Biol. 2024 Feb 27:106556. doi: 10.1016/j.biocel.2024.106556. Online ahead of print.ABSTRACTMitochondrial 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.PMID:38423381 | DOI:10.1016/j.biocel.2024.106556
Source: The International Journal of Biochemistry and Cell Biology - Category: Biochemistry Authors: Source Type: research