Article

Identification and Characterization of a Small Molecule Ligand for the Huntingtin-HAP40 Complex

2025-11-21

Abstract excerpt

Huntington’s disease (HD) is caused by a CAG repeat expansion mutation, giving rise to a polyglutamine expansion in the huntingtin (HTT). However, the explicit molecular functions of HTT and opportunities for direct pharmacological modulation remain incompletely understood. Here, we report the discovery of a small molecule ligand for the full-length HTT protein in complex with its partner, HAP40. Using affinity se...

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Literature Corpus work: bff39093-843e-5770-aa14-345caaa7a1bb
DOI: 10.1101/2025.11.21.689733

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