UK Launches MRC Centre of Research Excellence to Pioneer Mitochondrial Genome Therapeutics
核心洞察
A new MRC Centre of Research Excellence in Mitochondrial Genome Therapeutics (搜索) has been established, led by the University of Cambridge with partners across the UK, Europe, and Australia.
The centre aims to define how mitochondrial DNA (搜索) mutations cause disease and translate that knowledge into new therapeutic approaches for conditions affecting around 1 in 5,000 people.
The University of Manchester team will develop next-generation base editing tools to selectively target the most common disease-causing mtDNA mutations.
A major new collaborative research centre has been established to investigate how mutations in mitochondrial DNA (搜索) (mtDNA) cause disease and to accelerate the development of much-needed therapies. The MRC Centre of Research Excellence (CoRE) in Mitochondrial Genome Therapeutics, led by the University of Cambridge, brings together leading experts from across disciplines and continents to tackle a group of genetic disorders that currently have no cure.
Mitochondria provide the energy that cells need to function. Mutations in their DNA can cause serious, progressive conditions affecting organs and tissues with high energy demands, including the brain, heart and muscles. Around one in 5,000 people is affected by a mitochondrial disease (搜索), and beyond inherited conditions, mtDNA mutations are increasingly linked to neurodegeneration, metabolic disease, cardiovascular failure and age-related deterioration.
A Multi-Institutional Effort
The centre includes partners at the universities of Birmingham, Manchester, Heidelberg and Queensland, the Imagine Institute in Paris, patient charity The Lily Foundation (搜索), and industry organisations worldwide. By bringing together academic, clinical, patient and industry perspectives, the centre aims to establish a long-term research platform that can define the causes of mitochondrial disease (搜索) and accelerate progress towards therapies.
Professor Michal Minczuk of the University of Cambridge, the project lead, commented on the wider collaboration: "We will be building a long-term UK research platform with the scale, expertise and infrastructure needed to position the UK as a global leader in mitochondrial genome therapeutics. Our goal is to create the scientific foundations that will enable entirely new therapeutic strategies and offer renewed hope to patients and families affected by mitochondrial disease (搜索)."
Manchester's Base Editing Approach
The Manchester team, led by Sarah Lovelock, Professor of Biological Chemistry in the Department of Chemistry and the Manchester Institute of Biotechnology, will combine genome mining, computational enzyme design and laboratory evolution to develop next-generation base editing tools capable of selectively targeting the most common disease-causing mutations in mitochondrial DNA (搜索). This approach represents a cutting-edge strategy for correcting pathogenic mtDNA mutations at their source.
Birmingham's Expertise in Mitochondrial Biology
The University of Birmingham contributes unique models of mitochondrial disease (搜索), expertise in mitochondrial biology, and world-leading metabolic analysis platforms to the research programme. Professor Daniel Tennant, Professor of Biochemistry from the Department of Metabolism and System Science in the College of Medicine and Health, explained: "The significant impact families affected by mitochondrial disease face demonstrates the need for more research in this area. We are delighted to join the new MRC CoRE and work together to provide pioneering approaches to understanding and treating diseases caused by mutations in the mitochondrial genome."
Dr Sofia Ahola from the School of Medical Sciences and Professor Hansong Ma from the School of Biosciences also join the effort from Birmingham.
Building on UK Leadership
The MRC CoRE will harness emerging technologies to engineer the mitochondrial genome, delivering advanced models of prevalent pathogenic mtDNA mutations. The initiative builds on the UK's established leadership in mitochondrial science, including the development of mitochondrial replacement therapy which prevents the inheritance of mtDNA mutations.
Dr Ceri Williams, Executive Director of Challenge Led Themes at MRC, said: "The UK has been at the helm of mitochondrial science, having led on the development of mitochondrial replacement therapy which prevents the inheritance of mtDNA mutations. We are delighted to announce this new MRC CoRE, which builds on these foundations to bring together expertise from around the world and across sectors to make real progress towards understanding the root causes of mtDNA mutations."
She added: "Taking an interdisciplinary approach to tackle these challenges has the potential to radically transform health research in this field, improving prevention, detection and treatment, boosting outcomes for patients and protecting families affected by the disease."
The Lily Foundation (搜索)'s involvement will ensure that the priorities of families with lived experience have the chance to shape the research going forward, grounding the scientific endeavour in the real-world needs of those affected by mitochondrial disease (搜索).