U.S., May 14 -- ClinicalTrials.gov registry received information related to the study (NCT06967831) titled 'Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells' on April 20.
Brief Summary: In this project, the investigators are using iPSC lines derived from patients with Leigh syndrome that carry mutations in the mitochondrial (mtDNA) and in the nuclear DNA (nDNA) to reprogram them into neural progenitor cells and into dopaminergic neurons. The researchers are using this experimental system to screen FDA (Food and Drug Administration, USA) and EMA (European Medicines Agency) approved drugs for a positive effect on Leigh patient-derived neuronal cells (drug repurposing) using various biochemical, optic, and morphol...