The Medical Research Council has invested £50 million in a new centre aimed at improving understanding and treatment of mitochondrial diseases, a group of genetic disorders that affect around one in 5,000 people and currently have no cure.
The new MRC Centre of Research Excellence in Mitochondrial Genome Therapeutics will bring together experts from across disciplines to define how mutations in mitochondrial DNA cause disease and then translate that knowledge into therapies. The centre is led by the University of Cambridge and will work with the University of Birmingham, the University of Manchester, Heidelberg University, the University of Queensland and the Imagine Institute in Paris.
Why mitochondrial disease remains such a major challenge
Mitochondrial dysfunction can lead to severe disability, progressive decline and premature death. According to the MRC, mutations in mitochondrial DNA are also increasingly linked to neurodegeneration, metabolic disease, cardiovascular failure and age-related deterioration, widening the potential impact of the research beyond inherited mitochondrial disorders alone.
Professor Michal Minczuk, the project lead at the University of Cambridge, said the centre will provide new approaches to understanding and treating diseases caused by mutations in the mitochondrial genome. He added that the goal is to build a long-term UK research platform with the scale, expertise and infrastructure needed to position the country as a global leader in mitochondrial genome therapeutics.
Collaboration and patient involvement at the centre of the programme
The MRC said the project will harness emerging technologies to engineer the mitochondrial genome and create advanced models of pathogenic mtDNA mutations. It will also involve leading charities, including the Lily Foundation, and industry partners worldwide.
Dr Ceri Williams, Executive Director of Challenge Led Themes at the MRC, said the UK has long played a leading role in mitochondrial science and that the new centre builds on that foundation to advance prevention, detection and treatment. Liz Curtis, chief executive and founder of the Lily Foundation, said the initiative reflects the kind of strategic, collaborative research that can help shape future treatments while embedding patient priorities from the outset.
The investment is intended to support the scientific foundations for entirely new therapeutic strategies and offer renewed hope to patients and families affected by mitochondrial disease.
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