Article

Structure-function dissection of huntingtin exon 1 identifies a PRD-driven modifier of neuronal toxicity in Huntington’s disease

2026-07-27

Abstract excerpt

The Huntingtin gene ( HTT ) contains a conserved, yet expandable CAG repeat within exon 1. While the pathogenic expansion in Huntington’s Disease (HD) is well studied, the role of surrounding domains remains unclear. Using genome-edited mini-organoids and neurons, we dissected HTT exon 1 and found species-specific toxicity: the human variant caused more severe deficits than the mouse. Swapping the proline-rich...

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Literature Corpus work: 68f564cd-7bc4-55a0-b2de-43d765dc2ad4
DOI: 10.64898/2026.07.23.740298

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