Open access
Sep 2026
Brain region-resolved pharmacodynamics of an antisense oligonucleotide targeting human SNCA 3′UTR in BAC-hSNCA rats
Alpha-synuclein accumulation contributes to Parkinson’s disease (PD), and lowering α-synuclein expression is a candidate disease-modifying approach. The brain-wide pharmacodynamic profile of human-targeting antisense oligonucleotides (ASOs) remains incompletely defined in humanized models that preserve the regulatory a...
Marianna Naki, F. Kattan, M. Pantazopoulou et al.
· Translational Neurodegenerat... · 0 citations