Congratulations to Professor Judy Hirst, who has been awarded an Advanced Grant from the European Research Council (ERC). Judy's grant is for the energy-converting mechanism of a modular biomachine: U...

Fifty years since its discovery, scientists have finally worked out how a molecular machine found in mitochondria, the ‘powerhouses’ of our cells, allows us to make the fuel we need from s...

imageimageEfficient mitochondrial gene therapy is achieved with a novel tandem zinc finger nuclease (mtZFN) architecture delivered via adeno-associated viral (AAV) vectors, significantly reducing mutation loads in muscle tissue and rescuing ...

Congratulations to Kasparas Petkevicius, who has been awarded a grant of $50,000 from the Batten Disease Global Research Initiative, which will fund a research project “Exploring the efficacy of bis(m...

Rare disease research faces unique challenges including lack of investment, small patient populations, fragmented research and challenging routes to…