Aug 2026
Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington's disease pathogenesis in mice.
These findings position somatic expansion as a promising therapeutic target and demonstrate the potential of RNAi-based cosilencing of MSH3 and HTT as a disease-modifying strategy for HD.
Jillian Belgrad, Ashley Summers, C. Landles et al.
· Science Translational Medici... · 0 citations