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Lowering mutant huntingtin by small molecules relieves Huntington's disease symptoms and progression.

EMBO molecular medicine - 1 Mar 2024

Bahat Anat, Itzhaki Elad, Weiss Benjamin, Tolmasov Michael, Tsoory Michael, Kuperman Yael, Brandis Alexander, Shurrush Khriesto A, Dikstein Rivka

Abstract excerpt

Huntington's disease (HD) is an incurable inherited disorder caused by a repeated expansion of glutamines in the huntingtin gene (Htt). The mutant protein causes neuronal degeneration leading to severe motor and psychological symptoms. Selective downregulation of the mutant Htt gene expression is considered the most promising therapeutic approach for HD. We report the identification of small molecule inhibitors...

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PMID: 38374466
DOI: 10.1038/s44321-023-00020-y
PMCID: PMC10940305

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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