Lowering the HTT1a transcript as an effective therapy for Huntingtons disease
Aikaterini Smaragdi Papadopoulou, Julia F. Alterman, Christian Landles, Edward J. Smith, Faith Conroy, Jemima M Phillips, María Cañibano-Pico, Iulia M Nita, Georgina F Osborne, Arzo Iqbal, Sarah G. Aldous, Marie K. Bondulich, Casandra Gomez-Paredes, Kirupa Sathasivam, Daniel O’Reilly, , Gillian P. Bates
The paper shows that lowering HTT1a transcript levels delays molecular and neuropathological phenotypes in a Huntington's disease mouse model.
bioRxiv · Jun 10, 2025 · PreprintNeuroscience