Utilizing patient-derived neurons, Scientists at Northwestern Medicine are trying to come up with a brand new strategy. This is for the treatment of Parkinson’s disease through alleviation of harmful effects of genetic mutation. The study was published on Science Translational Medicine.
Some of the experimental treatments that focused on treating genetic disorders targeted mutated enzymes or proteins. However, this particular research took an entirely different approach toward the treatment of Parkinson’s disease. In this study, the researchers made an attempt to enhance the capabilities of the healthy enzymes as opposed to efforts to repair the broken ones. This new approach has successfully reduced the symptoms of Parkinson’s disease. The approach is studied in both mouse models and in the brain cells of human beings.
The New Study Focuses on the Importance of Patient-Derived Neurons
According to this new study, in case of Parkinson’s disease, mutations in the GBA1 gene pose a serious genetic risk factor for the development of the disease. The study also mentions that GBA1 codes of an enzyme named glucocerebrosidase or GCase is significant for the proper functioning of neuronal. Parkinson’s disease related mutations are capable of rendering GBA1 useless. This can lead to build-up of harmful proteins in the dopamine-producing neurons. With the reducing population of neurons, the patient witnesses various symptoms like slow movements and tremors. However, a few of the medicines can provide some relief from symptoms. However, there is no stopping to the progression of this disease.
Dr. Dimitri Krainc heads the neurology department, Center for Neurogenetics, Northwestern University Feinberg School of Medicine. Dr. Krainc opined that the research underscores the activation of wild-type GCase as a probable therapeutic target for many different forms of Parkinson’s disease.
Another study headed by Dr. Krainc and published in the journal Science throws light on pathological characteristics of Parkinson’s disease human neurons only and not in mouse. This strengthens the importance of this new study.