Article

Development of a novel VHH intrabody targeting the N17 region of huntingtin exon 1 protein that prevents inclusion body formation

2026-04-13

Abstract excerpt

Huntington’s disease (HD) is a progressive neurodegenerative disease caused by a mutation in the exon 1 of the huntingtin ( HTT ) gene, which leads to an extended polyglutamine (polyQ) tract in the mutant protein. As a result, mutant huntingtin (mHTT) exon 1 fragments aggregate in cells, which disrupts proper neuronal function and eventually induces cell death. The selective reduction of these toxic mHTT fragment...

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Literature Corpus work: cbe23b14-cdcc-5818-95a7-3aed389a1ede
DOI: 10.64898/2026.04.09.716913

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