NeurosciencebioRxivPreprint
VO659, a CAG repeat-targeting antisense oligonucleotide, lowers multiple neurotoxic mutant huntingtin proteins in Huntington's disease
Bas Röttgering, Rudie Weij, Suzanne Bijl…·Nicole A. Datson
VO659 reduces multiple neurotoxic mutant huntingtin proteins in Huntington's disease by targeting CAG repeats, while sparing wild-type HTT.
DOI 10.64898/2026.10.16.758452