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This year's Nobel Prize in Medicine and Physiology recognizes the discovery of the importance of microRNA (miRNA) in gene regulation - a finding that has fundamentally changed modern medicine. Researchers Victor Ambros and Gary Ruvkun discovered that these small RNA molecules act as gene regulators and control essential biological processes. Their work opens up new possibilities for medicine and paves the way for personalized therapeutic approaches of the future.
The Discovery and Function of microRNAs
MicroRNAs are small, non-coding RNA molecules that are formed in the cell nucleus and enter the cytoplasm after several processing steps. There they bind to messenger RNAs (mRNAs) and influence their stability and translation, thereby regulating the production of specific proteins. This enables miRNAs to regulate hundreds of genes simultaneously and control complex processes such as cell division and cell death (see diagram below).
The discovery of miRNAs in 1993 by Victor Ambros and Gary Ruvkun marked a paradigm shift: unlike mRNAs, which carry genetic information for protein synthesis, miRNAs have a regulatory function and decide which mRNAs are actually translated into proteins. This insight opened up a new dimension of gene regulation and laid the foundation for modern therapies that act specifically at the RNA level and offer the potential for personalized medicine.
Therapeutic Potential of miRNAs
The understanding of the regulatory function of miRNAs has enriched the field of therapeutic development with new approaches. Two main strategies offer promising options for the treatment of serious diseases:
miRNA antagonists (antagomirs): These compounds aim to block disease-promoting miRNAs and thus reduce their negative impact on specific gene networks.
miRNA mimetics: These molecules mimic missing or deficient miRNAs to restore normal gene regulation.
The first miRNA-based therapies are in clinical trials and show potential in the treatment of cancer, autoimmune diseases and rare genetic disorders. The ability of miRNAs to modulate entire gene networks represents a unique approach to influence genetic diseases at the source.
Current State of Research and Areas of Application
ent years, advances in miRNA research have prompted leading biotechnology companies and research institutes to invest specifically in miRNA-based therapies. The main areas of application for these new therapies include:
Oncology: miRNA profiles are critical in various cancers and are being used to specifically inhibit tumor growth. Therapeutic approaches such as MRX34 (developed by Mirna Therapeutics), for example, mimic the tumor suppressor function of miR-34a.
Cardiovascular diseases: miRNAs regulate crucial processes in the cardiovascular system. Companies such as miRagen Therapeutics are developing miRNA therapies that could reduce the risk of cardiovascular complications.
Neurology: Preclinical studies indicate that miRNAs have neuroprotective effects by degrading harmful proteins and promoting the regeneration of neuronal cells.
Companies such as Regulus Therapeutics, Alnylam Pharmaceuticals and Moderna are driving research and development in this area, while also working to establish miRNAs as biomarkers for diagnosis and disease progression monitoring, as they can reflect specific disease patterns in the blood.
Challenges and Future Prospects
Although the potential of miRNA therapy is great, scientists still face several challenges. One of the biggest hurdles is the targeted delivery of the miRNA molecules to the affected cells in the body. Researchers are working on carrier systems to ensure that the miRNA reaches the right target cells and exerts its effect there. Another obstacle is the potential side effects of manipulating gene regulation. Intensive research is needed to minimize these risks and ensure the safety of the therapies.
Conclusion
MiRNA-based therapies are among the most innovative and promising approaches in modern research and have the potential to profoundly change medicine. Despite existing challenges, companies and scientists around the world are working hard to bring the first safe and effective applications to market. The recent Nobel Prize honor for the discovery of the importance of miRNA and its role in gene regulation underscores the relevance of these small but powerful molecules. The future of miRNA therapy looks promising - and could fundamentally change the way many diseases are treated.
Sources:
MicroRNA: Therapieoptionen in der Pipeline | APOTHEKE ADHOC
Nobelpreis für Medizin: microRNA in der Medizin - [GEO]
Medizin-Nobelpreis geht an microRNA-Entdecker Ambros und Ruvkun | tagesschau.de
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