Article
Allele-selective lowering of mutant HTT protein by HTT-LC3 linker compounds.
Nature - 1 Nov 2019
Li Zhaoyang, Wang Cen, Wang Ziying, Zhu Chenggang, Li Jie, Sha Tian, Ma Lixiang, Gao Chao, Yang Yi, Sun Yimin, Wang Jian, Sun Xiaoli, Lu Chenqi, Difiglia Marian, Mei Yanai, Ding Chen, Luo Shouqing, Dang Yongjun, Ding Yu, Fei Yiyan, Lu Boxun
Abstract excerpt
Accumulation of mutant proteins is a major cause of many diseases (collectively called proteopathies), and lowering the level of these proteins can be useful for treatment of these diseases. We hypothesized that compounds that interact with both the autophagosome protein microtubule-associated protein 1A/1B light chain 3 (LC3)1 and the disease-causing protein may target the latter for autophagic clearance. Mutant...
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