Prof. Matthis Synofzik, head of the Section “Translational Genomics of Neurodegenerative Diseases” at HIH, is considered one of the pioneers of a medical revolution: RNA therapy. This therapy allows genetic defects to be corrected directly at their source - an approach that offers hope for patients with rare neurological diseases, but also holds potential for widespread diseases such as Alzheimer's.

Around 80 percent of rare diseases are genetic, but there is currently no treatment available for 95 percent of those affected. Synofzik's team uses antisense oligonucleotides (ASOs) – tiny RNA molecules that specifically “overwrite” diseased genes. In a seven-year-old patient with ataxia teleangiectasia, this approach slowed the progression of the disease.

“We attack the disease process at its root,” explains Synofzik. Instead of merely alleviating symptoms, RNA medicine can correct the actual genetic defect. His goal is to standardize the therapeutic process in order to make customized RNA drugs available to many patients more quickly and cost-effectively.

Phase 2 studies are also currently underway for Alzheimer's disease – initial results show that the disease-causing tau proteins in the brain decrease significantly after RNA treatment.

RNA therapies are still in their infancy. But experts agree: they mark the beginning of a new era of personalized medicine – and could soon give hope to many people for whom there has been no treatment to date.

You can download the article from FOCUS Gesundheit here (in German)

Foto: Prof. Matthis Synofzik

Copyright: UKT