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Editing Better Muscles

Maximising gene editing to repair the mutated dystrophin gene responsible for Duchenne muscular dystrophy

03 April 2019

Editing Better Muscles

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease caused by mutations to dystrophin – a protein inside muscle cells that provides essential mechanical stability. Without functional dystrophin, DMD sufferers gradually lose muscle strength, become increasingly frail, have trouble breathing and generally don't live past their 30s. The nature of the dystrophin mutations is such that gene-editing technology has been posited as a potential treatment. Indeed, engineered mice and dogs carrying DMD mutations have had functional dystrophin partly restored as a result of gene editing. Scientists are now optimising the technique to maximise its efficiency in human cells. The image shows heart cells derived from reprogrammed DMD patient cells before (left) and after (right) gene editing to restore dystrophin (red). These technical successes in mammalian models and patient cells brings the promise of a gene-editing treatment for DMD one step closer to reality.

Written by Ruth Williams

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BPoD stands for Biomedical Picture of the Day. Managed by the MRC Laboratory of Medical Sciences until Jul 2023, it is now run independently by a dedicated team of scientists and writers. The website aims to engage everyone, young and old, in the wonders of biology, and its influence on medicine. The ever-growing archive of more than 4000 research images documents over a decade of progress. Explore the collection and see what you discover. Images are kindly provided for inclusion on this website through the generosity of scientists across the globe.

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