Allele-selective lowering of mutant HTT protein by HTT–LC3 linker compounds

@article{Li2019AlleleselectiveLO,
  title={Allele-selective lowering of mutant HTT protein by HTT–LC3 linker compounds},
  author={Zhaoyang Li and Cen Wang and Ziying Wang and Chenggang Zhu and J. Li and Tian Sha and Lixiang Ma and Chao Gao and Y. Yang and Yimin Sun and J. Wang and Xiaoli Sun and Chenqi Lu and M. DiFiglia and Y. Mei and Chen Ding and S. Luo and Yongjun Dang and Yu Ding and Yiyan Fei and B. Lu},
  journal={Nature},
  year={2019},
  volume={575},
  pages={203-209}
}
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 huntingtin protein (mHTT) contains an expanded polyglutamine (polyQ) tract and… Expand
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