Therapeutic validation of MMR-associated genetic modifiers in a human ex vivo model of Huntington's disease
Ross Ferguson, Robert Goold, Lucy A. Coupland, Michael D. Flower, Sarah J. Tabrizi
This study shows that reducing MMR gene expression in HD iPSCs slows CAG repeat expansion, suggesting these genes as therapeutic targets for HD.
bioRxiv · Dec 5, 2023 · PreprintNeuroscience