Screening for Therapeutics against Untreatable Mitochondrial Disorders: Using Neural Progenitors from Patient - iPSCs

摘要

Mitochondrial DNA (mtDNA) mutations cause untreatable disorders affecting the nervous system. These diseases suffer from a lack of viable modeling tools due to the challenges associated with mtDNA engineering. We recently showed that neural progenitor cells (NPCs) differentiated from patient-derived induced pluripotent stem cells (iPSCs) can be used to model mtDNA disorders and to set up phenotypic compound screenings. This strategy opens the way to the discovery of novel therapeutics against these so far untreatable human diseases.