Systems approaches to understanding mitochondrial function and dysfunction in disease

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Mitochondria produce the energy for our bodies. Defects in this process cause mitochondrial disease, which affects at least 1/4300 people. Diagnosis is often inconclusive and few if any effective treatments exist. State of the art CRISPR gene-editing tools will be used to make disease models mimicking the different types of mitochondrial disease. These will be used to understand how mitochondria function, identify new disease genes and develop new drugs.