Review
Open access
Oct 2026
Directing mutant huntingtin towards degradation: Strategies and therapeutic opportunities.
Huntington's Disease (HD) is a progressive neurodegenerative disease that is caused by a CAG-repeat expansion in the exon 1 of the huntingtin (HTT) gene, which results in the formation of toxic mutant HTT (mHTT) exon 1 fragments with expanded polyglutamine (polyQ) stretch. These mHTT fragments are prone to misfolding a...
Florence D. M. Wavreil, S. Schipper-Krom, E. Reits
· Journal of Huntington's Dise... · 0 citations