Gene therapy in Parkinson's disease: targeting the endplasmic reticulum proteostasis network

摘要

Parkinson’s disease(PD)is the second most common neurodegenerative disease affecting 1%of the population over 60 years of age.The progressive degeneration of dopaminergic neurons at the substantia nigra pars compacta(SNpc)results in a severe and gradual depletion of dopamine content in the striatum,a phenomena that is responsible for the characteristic motor symptoms of this disease.There is no cure for PD and available treatments only aim to restore dopamine deficits.Administration of the dopamine precursor Levodopa(L-DOPA)is the main temporal palliative treatment that increases overall dopamine levels.However,its chronic use limits its effectiveness and generates a number of adverse effects such as debilitating dyskinesia.