Autophagosome-tethering compounds selectively lower mutant ataxin-3 in preclinical models of spinocerebellar ataxia type 3
Hongyu Yuan, Zhao Chen, Xiaokai Shen, Yiqing Gong, Yan Tan, Jiawei He, Na Wan, Chunrong Wang, Linlin Wan, Linliu Peng, Lang He, Huirong Peng, Rong Qiu, Beisha Tang, Boxun Lu, Hong Jiang
Small-molecule autophagosome-tethering compounds selectively lower mutant ataxin-3 through an LC3- and autophagy-dependent mechanism and improve disease-related phenotypes in preclinical models of SCA3.
bioRxiv · Oct 8, 2026 · PreprintNeuroscience