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A pathogenic proteolysis-resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function.
Kim H, Lenoir S, Helfricht A, Jung T, Karneva ZK, Lee Y, Beumer W, van der Horst GB, Anthonijsz H, Buil LC, van der Ham F, Platenburg GJ, Purhonen P, Hebert H, Humbert S, Saudou F, Klein P, Song JJ. Kim H, et al. Among authors: karneva zk. JCI Insight. 2022 Sep 8;7(17):e154108. doi: 10.1172/jci.insight.154108. JCI Insight. 2022. PMID: 35943803 Free PMC article.