Rare hereditary disease: a small messenger molecule shows great effect
Brain researchers from Tübingen have developed a new approach for the treatment of hereditary spastic paraplegia (HSP). A gene therapy may treat the rare neurological disease directly at its point of origin. The therapeutic approach aims to treat the subtype spastic paraplegia gene type 5, or SPG5 in short. In this case, a defective gene leads to an increase of certain metabolic products in the brain that have a fatal effect on nerve cells. As a consequence, the affected patients lose their ability to walk in the long term. Now, the genetic defect has been able to be successfully circumvented for the first time in an animal model by repeatedly administering a messenger molecule (mRNA) into the blood. This led to a normalization of the concentration of neurotoxic metabolic products in the brain, reports an international research team led by Professor Schöls in collaboration with the company CureVac.
