Antisense Oligonucleotide–Mediated MSH3 Suppression Reduces Somatic CAG Repeat Expansion in Huntington’s Disease iPSC–Derived Striatal Neurons

Investigators showed that an MSH3-targeting antisense oligonucleotide effectively engaged with its RNA target in induced pluripotent stem cell (iPSC)–derived striatal neurons obtained from a patient with Huntington’s disease carrying 125 huntingtin CAG repeats.