Discovery of Small Molecules that Induce the Degradation of Huntingtin

Shusuke Tomoshige, Sayaka Nomura, Kenji Ohgane, Yuichi Hashimoto, Minoru Ishikawa

研究成果: ジャーナルへの寄稿学術論文査読

82 被引用数 (Scopus)

抄録

Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder caused by the aggregation of mutant huntingtin (mHtt), and removal of toxic mHtt is expected to be an effective therapeutic approach. We designed two small hybrid molecules (1 and 2) by linking a ligand for ubiquitin ligase (cellular inhibitor of apoptosis protein 1; cIAP1) with probes for mHtt aggregates, anticipating that these compounds would recruit cIAP1 to mHtt and induce selective degradation by the ubiquitin-proteasome system. The synthesized compounds reduced mHtt levels in HD patient fibroblasts and appear to be promising candidates for the development of a treatment for HD.

本文言語英語
ページ(範囲)11530-11533
ページ数4
ジャーナルAngewandte Chemie - International Edition
56
38
DOI
出版ステータス出版済み - 2017 9月 11

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