Main idea: A new therapeutic method based on gene therapy found for Parkinson’s disease. It is based on short samples of DNA that encodes α-synuclein, one of the crucial regulators of Parkinson’s disease progression.
Parkinson’s disease (PD) is a prevalent neurodegenerative disease with no approved disease-modifying therapies. Multiplications, mutations in the SNCA gene either cause or increase risk for PD. Antisense oligonucleotides (ASOs) reduce the production of aSyn in rodent models of PD. Reduced aSyn production leads to the prevention and removal of established aSyn pathology and prevents dopaminergic cell dysfunction. In this study, we identify human SNCA-targeting ASOs that efficiently suppress the human S NCA transcript in vitro. We demonstrate broad activity and distribution of the human sNCA ASOs throughout the nonhuman primate brain. By inhibiting the production of aSyn, it may be possible to reverse established pathology.