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Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations.
Dulla K, Slijkerman R, van Diepen HC, Albert S, Dona M, Beumer W, Turunen JJ, Chan HL, Schulkens IA, Vorthoren L, den Besten C, Buil L, Schmidt I, Miao J, Venselaar H, Zang J, Neuhauss SCF, Peters T, Broekman S, Pennings R, Kremer H, Platenburg G, Adamson P, de Vrieze E, van Wijk E. Dulla K, et al. Among authors: buil l. Mol Ther. 2021 Aug 4;29(8):2441-2455. doi: 10.1016/j.ymthe.2021.04.024. Epub 2021 Apr 23. Mol Ther. 2021. PMID: 33895329 Free PMC article.
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: buil lc. JCI Insight. 2022 Sep 8;7(17):e154108. doi: 10.1172/jci.insight.154108. JCI Insight. 2022. PMID: 35943803 Free PMC article.
20 results