GENE THERAPY OF PARKINSON`S DISEASE

Modern methods of treatment of Parkinson's disease (PD) remain to be unsuccessful in cessation of the progression of neurodegenerative processes in the brain. The role of certain genes is proven by the etiology of PD, 10–15% of cases represent a monogenic disease, and in 85–90% of patients the disease is related with multifactorial pathology with the combination of internal and external factors involved in the pathological process. The discovery of the role of genetic factors and identification of several causative genes in the etiology of PD has provided a new look at the prospects of treatment. Gene therapy of PD is a good alternative to improve the survival and the preservation of the function of neurons that might help to reduce the adverse side effects associated with current methods of symptomatic therapy. The ability to influence the synthesis of dopamine was considered as the basic aim of the gene therapy to compensate for its deficit. However, over time the view of the problem has expanded considerably, in addition to an increase in the local concentration of dopamine, research is also providing neuroprotective actions on dopaminergic neurons undergoing degeneration, improvement of the microenvironment of dopaminergic/medium energy systems involved in the development of PD and normalization of genetic abnormalities of cells associated with PD. Depending on the route of influencing the gene there are used methods to turn off the abnormal gene or the introduction into cells of the patient a new gene to change his functionality. Research in the field of gene therapy of PD develops rapidly, and some of them have already enter the stage of clinical trials. This article presents an overview of the results of available studies, conducted over the past five years, the questions of the main directions of gene therapy of PD, achievements, and also briefly discussed difficulties of gene therapy of PD, on the way to achieving goals