A protein called DJ-1 plays a vital role in maintaining the function of nerve cells. If the body is unable to produce sufficient amounts of DJ-1 for any reason, the body’s important nerve cells die. The result of this will be the onset of neurodegenerative diseases such as Parkinson’s. Production of important proteins such as DJ-1 can be permanently disrupted or stopped if the genetic program or processes involved in producing these proteins are defective. In a new study, researchers took skin cell samples from Parkinson’s patients and reprogrammed them into induced pluripotent stem cells and differentiated them into nerve cells in laboratory conditions. These nerve cells are very similar to neurons in the affected areas of the brain.
Since it is not possible to directly isolate neurons from patients’ brains (for health and ethical reasons), cell reprogramming can be a way to evaluate the clinical characteristics of patients’ neurons in vitro. Using this method, they were able to identify one of the causes of the genetic form of Parkinson’s disease, in which the PARK7 gene is mutated. A precise bioinformatics algorithm also indicated the identification of potentially active substances for drug treatments. One of the substances identified for this genetic form of Parkinson’s disease was a combination of phenyl butyric acid and RECTAS. According to the researchers, the combination of these two active substances and their use in laboratory tests showed that they effectively increase the production of the important DJ-1 protein.