Abstract

a-Synuclein Targeted Antisense Oligonucleotides For Treatment of Parkinson’s Disease

Hideki Mochizuki, Professor and Chairman, Osaka University Graduate School of Medicine

Parkinson’s disease (PD) is a neurodegenerative disease characterized by the loss of dopaminergic neurons in the substantia nigra and the presence of Lewy bodies and neurites predominantly composed of aggregated a-synuclein (SNCA). We designed and synthesized amido-bridged nucleic acids (AmNA)-modified antisense oligonucleotides (ASOs) that targeted SNCA for SNCA downregulation endogenously. AmNA-ASO-SNCA is a promising therapeutic strategy for SNCA-associated pathology in PD. I will present you a new technique to introduce this compound to the brain with focused ultrasound.