NeurosciencebioRxivPreprint
Autophagosome-tethering compounds selectively lower mutant ataxin-3 in preclinical models of spinocerebellar ataxia type 3
Hongyu Yuan, Zhao Chen, Xiaokai Shen…·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.