Allele-Specific Targeting of Mutant Ataxin-3 by Antisense Oligonucleotides in SCA3-iPSC-Derived Neurons

The authors provided proof of principle that allele-specific lowering of polyQ-expanded ataxin-3 by selective antisense oligonucleotides was feasible and long lasting with sparing of wildtype ataxin-3 expression in a human cell culture model that was genetically identical to spinocerebellar ataxia type 3 (SCA3) patients.