Kourkouta E, Weij R, González-Barriga A, Mulder M, Verheul R, Bosgra S, Groenendaal B, Puoliväli J, Toivanen J, van Deutekom JCT, Datson NA, Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide Molecular therapy Nucleic acids17:601-614 2019 |