Revolution in medicine: RNA research brings hope against muscle loss!

Revolution in medicine: RNA research brings hope against muscle loss!

RNA research has undergone remarkable development in recent years, particularly in relation to the development of RNA vaccines against COVID-19. These technological advances have led to three Nobel Prizes being awarded in the last four years for work in RNA research. A central aspect of this research is the understanding that RNA molecules are not only responsible for protein production, but can also be involved in the development of diseases. The ability to act therapeutically on RNA has the potential to revolutionize medicine in the future.

CRISPR technology plays a crucial role here, opening up new avenues in RNA and genetic research. Messenger RNAs (mRNAs) in particular have now been well researched, but much remains to be clarified regarding non-coding RNAs. The NUCLEATE Cluster of Excellence aims to make a significant contribution to the further development of RNA research and to promote new approaches in drug development.

Growth and challenges in RNA research

Expectations for nucleic acid-based drugs are high. In the current medical landscape, there are three main classes of active ingredients: small molecules, biologicals and soon nucleic acid-based drugs. These innovative therapies could enable broader influence on genes and the RNA precursors of proteins, significantly improving the treatment of diseases. There are currently fewer than 3,000 approved active ingredients that only bind to around 600 of the 20,000 different proteins in the human body.

One of the greatest challenges in the development of RNA-based drugs lies in the targeted and undamaged delivery of these therapeutics to their site of action. Advances in targeted effects have already been made, such as an active ingredient that specifically targets a microRNA in immune cells. Research on nucleic acids is carried out not only at the Technical University of Munich (TUM), but also in other disciplines such as virology and bioinformatics. Particularly noteworthy is the CNATM consortium, which is unique in Germany and specializes in nucleic acid therapeutics and includes numerous companies and research institutions in Munich and Bavaria.

Munich is considered a center for innovation in nucleic acid research, supported by a high density of small and medium-sized companies working on this topic. Experts from areas such as immunology and chemical structure research make significant contributions to developments in RNA research.

CRISPR technology as a game changer for genetic diseases

A notable aspect of current research is the application of CRISPR/Cas9 technology, which acts as genetic scissors and is an important tool in biological research. This technique makes it possible to specifically switch off specific genes in an organism in order to study their functions. After initially being used successfully in bacteria and yeast, the potential is also evident in other species such as zebrafish, mice and plant models. The cost-effectiveness and simplified process compared to previous genome editing methods make CRISPR/Cas9 a preferred approach in genetic research.

A practical example of the use of CRISPR is the work of Simone Schnurer, a leading scientist at the Max Delbrück Center (MDC). Her focus is on hereditary muscle wasting, particularly muscular dystrophies, which are caused by mutations in various genes. Schnurer's team is developing a method for using mRNA as genetic scissors, which is proving promising. Unlike traditional DNA approaches, mRNA has the ability to degrade after one to two days, avoiding risks of integration into the genome.

Although the research is producing promising results, scientists like Schnurer face challenges in practical application, such as transporting the modified RNA to target cells. They are currently planning clinical studies to test these innovative therapies, even if they are initially carried out without any direct clinical benefit for patients.

Interdisciplinary collaboration in basic research is considered crucial to achieving further progress in drug development. Numerous institutions are striving to integrate the latest technologies, such as mRNA research, in order to fundamentally improve the possibilities for treating genetic diseases. This opens a new chapter in medicine that raises many hopes for the future.