Depleting prion protein using splice-switching small molecules
Bo Liu, Benjamin Jung Fair, Zhiling Kuang, Zhichao Tang, Junxing Zhao, Lixian Zhou, Qingyao Kong, Ani Solanki, Connor J. Kenny, James A. Mastrianni, Rui Zhao, Yang Li, Jingxin Wang
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.
bioRxiv · Jun 27, 2026 · PreprintBiochemistry