Wellcome Centre for Mitochondrial Research (360G-Wellcome-203105_Z_16_Z)

Our mission is to transform the lives of patients with mitochondrial disease To achieve this mission we will build on progress made since the start of the Centre in May 2012 and combine: Three core scientific research themes - Understanding clinical mitochondrial disease: we will expand our unparalleled cohort of mitochondrial patients through extensive deep phenotyping, identifying key disease mechanisms at a clinical (whole patient) and organ level, and developing models of specific clinical features to enable improved treatment for patients with mitochondrial disease. - Understanding the genetic mechanisms underlying mitochondrial disease: we will elucidate genetic factors responsible for mitochondrial disease, exploring the mechanisms underlying the tissue specificity that characterises clinical disease and delineating the pathways underlying mitochondrial translation. - Prevention and treatment of mitochondrial disease: we will optimise the prevention of mitochondrial disease, developing novel therapies targeted at the mitochondrial genome and identifying new compounds to treat mitochondrial disease. We will ensure that every patient with mitochondrial disease has the opportunity to be involved in a clinical trial. Training the next generation of clinical and basic mitochondrial scientists. Engaging with patients to evolve our research focus and with policy makers to ensure our research findings improves care for patients with mitochondrial disease.