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Year Number of Results
2010 1
2012 3
2013 2
2014 2
2016 2
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2019 3
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Page 1
Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C.
Kaltak M, de Bruijn P, Piccolo D, Lee SE, Dulla K, Hoogenboezem T, Beumer W, Webster AR, Collin RWJ, Cheetham ME, Platenburg G, Swildens J. Kaltak M, et al. Among authors: beumer w. Mol Ther Nucleic Acids. 2023 Feb 18;31:674-688. doi: 10.1016/j.omtn.2023.02.020. eCollection 2023 Mar 14. Mol Ther Nucleic Acids. 2023. PMID: 36910710 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: beumer w. JCI Insight. 2022 Sep 8;7(17):e154108. doi: 10.1172/jci.insight.154108. JCI Insight. 2022. PMID: 35943803 Free PMC article.
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: beumer w. 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.
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models.
Dulla K, Aguila M, Lane A, Jovanovic K, Parfitt DA, Schulkens I, Chan HL, Schmidt I, Beumer W, Vorthoren L, Collin RWJ, Garanto A, Duijkers L, Brugulat-Panes A, Semo M, Vugler AA, Biasutto P, Adamson P, Cheetham ME. Dulla K, et al. Among authors: beumer w. Mol Ther Nucleic Acids. 2018 Sep 7;12:730-740. doi: 10.1016/j.omtn.2018.07.010. Epub 2018 Jul 23. Mol Ther Nucleic Acids. 2018. PMID: 30114557 Free PMC article.
18 results