Correcting mutations in muscle stem cells using new gene-editing technology
A new technique for editing genes can be used to correct mutations in muscle stem cells and pave the way for the first possible cell therapy for genetic muscle disorders. The ECRC team, led by Professor Simone Spuler, has published its results in the journal "JCI Insight". Muscle stem cells enable our muscles to build and regenerate for a lifetime through exercise. However, when certain muscle genes are mutated, the opposite occurs. In patients with muscular dystrophy, the skeletal muscle begins to weaken in childhood. Suddenly these children can no longer run, play the piano or climb stairs, and often by the age of 15 they are dependent on a wheelchair. There is currently no therapy for this condition.
Now we can access the gene mutations of these patients using CRISPR-Cas9 technology. We care for more than 2,000 patients in the Charité outpatient clinic for muscle disorders and quickly recognized the potential of the new technology. "
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