Satoshi Okawa

Jul 12, 2024 | biorxiv.org | Carmen Menacho |Satoshi Okawa |Iris Alvarez-Merz |Annika Wittich

AbstractMitochondrial disease encompasses untreatable conditions affecting tissues with high energy demands. A severe manifestation of mitochondrial disease is Leigh syndrome (Leigh), which causes defects in basal ganglia and midbrain regions, psychomotor regression, lactic acidosis, and early death. We previously generated isogenic pairs of Leigh cerebral organoids and uncovered defects in neuromorphogenesis. Here, we leveraged on this disease feature to devise drug discovery pipelines.