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.
[Molecular Therapy-Nucleic Acids]
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