VO659, a CAG repeat-targeting antisense oligonucleotide, lowers multiple neurotoxic mutant huntingtin proteins in Huntington's disease
Bas Röttgering, Rudie Weij, Suzanne Bijl, Ruurd C. Verheul, Janwillem Testerink, Lodewijk J.A. Toonen, Robert M. Bragg, Jeffrey B. Carroll, Chantal Beekman, Nicole A. Datson
VO659 reduces multiple neurotoxic mutant huntingtin proteins in Huntington's disease by targeting CAG repeats, while sparing wild-type HTT.
bioRxiv · Oct 22, 2026 · PreprintNeuroscience