Zhichao Tang

This study shows that U1C and Helix H of U1 snRNA are essential for branaplam's effect on weak 5 splice site recognition, while risdiplam requires additional factors for activity.

This paper develops PMO-based RNA-degrading chimeras that target SNCA mRNA to reduce α-synuclein levels and prevent pathological aggregation in vitro and in vivo.

This study develops a small-molecule approach to reduce prion protein by modulating pre-mRNA splicing, achieving ~70% PrP reduction in cells and demonstrating synergistic effects with a Luc7L activator in mice.