Muscles

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.

Simone Spuler is looking to use stem cells and CRISPR to prevent the muscle tissue of patients with genetic muscle disorders from further deteriorating – or even to repair it. This could lead to the first therapeutic approaches for diseases that have up to now been considered incurable.

Every year in Germany, around seven boys are born with an incompletely formed urethra and urethral sphincter. These children often remain incontinent. ECRC researchers want to find out whether a stem cell transplant could help them. The trial has been awarded approximately €4.3 million in funding from the Federal Ministry of Education and Research (BMBF) and the Berlin Institute of Health (BIH) at Charité.

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".

Three oscillating proteins cause new muscle cells to emerge from muscle stem cells in a balanced manner. In a paper being published in the journal "Nature Communications", a team led by MDC researcher Carmen Birchmeier explains in detail how this process works.

A muscle fiber consists of just one cell, but many nuclei. A team at the MDC led by Professor Carmen Birchmeier has now shown just how varied these nuclei are. The study, which has been published in Nature Communications, can help us better understand muscle diseases such as Duchenne muscular dystrophy.

Setting out to identify all proteins that make up the sarcomere, the basic contractile unit of muscle cells, resulted in an unexpected revelation, providing experimental evidence that helps explain a fundamental mystery about how muscles work. The research was published in Nature Communications.

Muscle pain and cramps are common side effects of statins. As an MDC and Charité research group now reports in Scientific Reports, the cholesterol reducers actually influence thousands of genes in muscle cells – thereby hindering cell growth and division.

Scientists have discovered a new subtype of muscle stem cells. These cells have the ability to build and regenerate new muscles, making them interesting targets for the development of gene therapies.

Professor Simone Spuler's team has developed a method to isolate muscle stem cells in such a way that they retain their regenerative potential. Dr. Verena Schöwel was awarded the Science4Life prize for her concept of using this technology for patients suffering from muscle diseases.