PSC-based drug discovery of mitochondrial disorders: Neural cells from patient-derived iPSCs as a novel system for drug discovery of mtDNA disorders

During Targeting Mitochondria 2015, Dr. Alessandro Prigione, from the Max Delbrück Center (MDC) of Berlin, will discuss how to develop novel drug discovery pipelines for mitochondrial DNA (mtDNA) disorders using induced pluripotent stem cells (iPSCs). Viable neural model systems of mtDNA brain disease are lacking due to the challenges of engineering mtDNA, thus hindering the search for therapeutic options. Here, we demonstrate that iPSC-derived neural cells may maintain the parental mtDNA profile and exhibit mitochondrial-based metabolism. Using this system, neural-specific dysfunctions due to homoplasmic mtDNA mutations could be identified. We therefore propose iPSC-derived neural cells as a novel model system for the establishment of personalized phenotypic drug discovery for untreatable mtDNA disorders affecting the nervous system.

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