Gene Engineering

Some hereditary genetic defects cause an exaggerated immune response that can be fatal. Using the CRISPR-Cas9 gene-editing tool, such defects can be corrected, thus normalizing the immune response, as researchers led by Klaus Rajewsky from the Max Delbrück Center now report in “Science Immunology.”

The Max Delbrück Center wants to shape the future of medicine through innovation. But turning basic research into marketable products requires gaps to be bridged. This is achieved by the Technology Transfer Office – through funding schemes such as BOOST and Pre-GoBio. The winning projects for 2023 have now been selected.

Klaus Rajewsky’s life reads like a chronicle of immunogenetics. He grasped the opportunities offered by radical changes in molecular biology. He established standard methods for researching gene functions and diseases like cancer. And he continues to contribute to our understanding of antibody-producing B cells.

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.

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".

MDC researchers can now induce diverse genetic mutations and track their effects on a large scale in microscopic worms. The team describe their in vivo parallel genetics approach and new analysis software called crispr-DART in the journal "Cell Reports".

Harnessing jumping genes to advance gene-therapy cancer treatment: In an effort to optimize the Sleeping Beauty system, the MDCell – Helmholtz Innovation Lab is extending its collaboration with PlasmidFactory.

Finnish Artist Emilia Tikka will spend two months as Artist in Residence at the Max Delbrück Center for Molecular Medicine in Berlin to explore the genome editing technology CRISPR-Cas9. The unusual exchange between art and science will result in an exhibition at the Opening of STATE Studio Berlin.

The tissue biobank “MyoPax” was selected as one of six new business ideas that are going to be funded by Helmholtz. It will store patient's muscle stem cells until regenerative therapies are approved.

The conference “Muscle Development, Regeneration and Disease 2018” took place at the MDC in late April. This event has in recent years become an increasingly important international platform for muscle research.