A CAG repeat threshold for therapeutics targeting somatic instability in Huntington's disease
Sarah G. Aldous, Edward J. Smith, Christian Landles, Georgina F Osborne, María Cañibano-Pico, Iulia M Nita, Jemima M Phillips, Yongwei Zhang, Bo Jin, Marissa B. Hirst, Caroline Benn, Brian C Bond, Winfried Edelmann, Jonathan R. Greene, Gillian P. Bates
This study shows that reducing MSH3 in Huntington's disease mouse models prevents somatic CAG repeat expansion but does not accelerate disease onset, suggesting somatic expansion may not be required for pathogenesis.
Brain · Feb 21, 2024Peer-reviewedNeuroscience