Muscles

In neuromuscular diseases, neurons and muscle cells stop communicating properly. Researchers led by Mina Gouti can now model this in 2D in a culture dish. Writing about their findings in “Nature Communications”, they say the new model promises to revolutionize high-throughput drug screening studies.

The UNIPRENEURS Initiative has recognized the entrepreneurial spirit of 20 professors, among them ECRC researcher Simone Spuler. As a co-founder of the startup MyoPax, she is pioneering stem cell technology to develop regenerative therapies for previously incurable muscle diseases.

MyoPax develops regenerative therapies for previously incurable muscle diseases. An interview with co-founders Prof. Simone Spuler and Dr. Verena Schöwel-Wolf, now board member and CEO respectively

Muscle cells are very good at repairing damage. But if this fails, stem cells step in to regenerate the tissue. Carmen Birchmeier investigates the molecular signaling pathways that underlie these and other vital mechanisms.

To perform coordinated movements, we rely on special sensory neurons in our muscles and joints. Without them, the brain wouldn’t know what the rest of our body was doing. A team led by Niccolò Zampieri has studied their molecular markers to better understand how they work and describes the results in “Nature Communications”.

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.