Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntingtons disease pathogenesis in mice
Jillian Belgrad, Ashley Summers, Christian Landles, Jonathan R. Greene, Samuel R. Hildebrand, Emily G. Knox, Ellen Sapp, Nozomi Yamada, Raymond C. Furgal, Rachael Miller, Georgina F Osborne, Kathryn O. Chase, Eric Luu, Jason Freedman, Brianna M. Bramato, , Anastasia Khvorova
This study shows that combining RNAi to silence MSH3 and HTT reduces huntingtin inclusions and restores gene expression in HD mice.
bioRxiv · Jun 24, 2025 · PreprintNeuroscience