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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.
Translational Genomics Group (Dr. Artero’s Lab), a Spanish research laboratory affiliated to the University of Valencia (UVEG) and INCLIVA Biomedical Research Institute
Valencia, Spain (PRUnderground) January 18th, 2021
Translational Genomics Group (Dr. Artero’s Lab), a Spanish research laboratory affiliated to the University of Valencia (UVEG) and INCLIVA Biomedical Research Institute, is pioneer in discovering therapeutic targets and innovative drug candidates for rare diseases, has announced the research on the development of novel DM1 (Myotonic Dystrophy Type 1) therapies based on recent new knowledge on microRNA class molecules. Being the most common muscular dystrophy in adults, affecting around 90,000 people in Europe and 75,000 in the US, the researchers from the Translational Genomics Group announced that their DM therapy could help these people and improve their quality of life.