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/PRNewswire/ -- MilliporeSigma, the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany, is the first CTDMO (contract testing, development...
The successful development of the mRNA active ingredient highlights the vital role basic research plays in applications.
Mutations that lead to muscle atrophy can be repaired with the gene editor CRISPR-Cas9. A team led by ECRC researcher Helena Escobar has now introduced the tool into human muscle stem cells for the first time using mRNA, thus discovering a method suitable for therapeutic applications.
Correcting mutations in muscle stem cells using new gene-editing technology A new gene-editing technique can be used to correct mutations in muscle stem cells, paving the way for the first potential cell therapy for genetic muscle disorders. The ECRC team led by Professor Simone Spuler have published their findings in the journal "JCI Insight". Muscle stem cells enable our muscle to build up and regenerate over a lifetime through exercise. But if certain muscle genes are mutated, the opposite occurs. In patients suffering from muscular dystrophy, the skeletal muscle already starts to weaken i...
E-Mail IMAGE: The confocal microscope image shows the patient's primary muscle stem cells, which have continued to proliferate after repair of the mutation using "base editing ". view more Credit: Spuler Lab, ECRC Muscle stem cells enable our muscle to build up and regenerate over a lifetime through exercise. But if certain muscle genes are mutated, the opposite occurs. In patients suffering from muscular dystrophy, the skeletal muscle already starts to weaken in childhood. Suddenly, these children are no longer able to run, play the piano or climb the stairs, and often they are ...