Validation of the Rip Kinase Pathway as a Therapeutic Target in Parkinson's Disease
Target Validation, 2015
A genetic connection has been shown over the past few years between Parkinsonís and Gaucherís diseases, such that people with genetic mutations that cause Gaucherís disease have a higher statistical risk of developing Parkinsonís disease, and some people with Parkinsonís have the genetic mutation that causes Gaucherís disease. However, at this stage, researchers are unable to explain the molecular connection between these two diseases.
Our study tests the hypothesis that there may be common biochemical pathways that link these two diseases. In particular, we will test whether a pathway (the so-called ĎRipkí pathway), which we recently discovered to be involved in brain pathology in Gaucherís disease, is also activated in Parkinsonís disease.
We will do this by a set of biochemical experiments in which we examine components of the Ripk pathway in human brain tissues that we obtained from colleagues in London from people with Parkinsonís disease, Gaucherís disease, and with both Parkinsonís and Gaucherís diseases. We will next induce Parkinsonís disease in a pre-clinical model that is defective is some components of the Ripk pathway to determine whether eliminating this pathway improves Parkinsonís symptoms.
Impact on Diagnosis/Treatment of Parkinsonís Disease:
If this approach is successful, it might indicate that drugs targeted to the Ripk pathway could act as novel therapeutic targets for Parkinsonís disease. Although such drugs are not available at the moment, the involvement of the Ripk pathway in a number of other human diseases is stimulating many drug companies to attempt to develop drugs directed to this pathway.
This project was selected for a Stern Discovery Award with support from the former Michael Stern Parkinson's Research Foundation, which merged with The Michael J. Fox Foundation in 2015.
Professor at Weizmann Institute of Science
Location: Rehovot, Israel