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Discovery of Small Molecule Inhibitors of Huntingtin Exon 1 Aggregation by FRET-Based High-Throughput Screening in Living Cells.

ACS chemical neuroscience - 5 Aug 2020

Lo Chih Hung, Pandey Nitin K, Lim Colin Kin-Wye, Ding Zhipeng, Tao Meixin, Thomas David D, Langen Ralf, Sachs Jonathan N

Abstract excerpt

Huntington's disease (HD) is the most common inherited neurodegenerative disorder and one of the nine polyglutamine (polyQ) diseases. HD is characterized by the pathological aggregation of the misfolded huntingtin exon 1 protein (Httex1) with abnormally long polyQ expansion due to genetic mutation. While there is currently no effective treatment for HD, inhibition of aggregate formation represents a direct...

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PMID: 32568514
DOI: 10.1021/acschemneuro.0c00226

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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