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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: swildens j. 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.
QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study.
Bornert O, Hogervorst M, Nauroy P, Bischof J, Swildens J, Athanasiou I, Tufa SF, Keene DR, Kiritsi D, Hainzl S, Murauer EM, Marinkovich MP, Platenburg G, Hausser I, Wally V, Ritsema T, Koller U, Haisma EM, Nyström A. Bornert O, et al. Among authors: swildens j. J Invest Dermatol. 2021 Apr;141(4):883-893.e6. doi: 10.1016/j.jid.2020.08.018. Epub 2020 Sep 16. J Invest Dermatol. 2021. PMID: 32946877 Free article.
Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis.
Beumer W, Swildens J, Leal T, Noel S, Anthonijsz H, van der Horst G, Kuiperij-Boersma H, Potman M, van Putten C, Biasutto P, Platenburg G, de Jonge H, Henig N, Ritsema T. Beumer W, et al. Among authors: swildens j. PLoS One. 2019 Jun 28;14(6):e0219182. doi: 10.1371/journal.pone.0219182. eCollection 2019. PLoS One. 2019. PMID: 31251792 Free PMC article.
Quaking, an RNA-binding protein, is a critical regulator of vascular smooth muscle cell phenotype.
van der Veer EP, de Bruin RG, Kraaijeveld AO, de Vries MR, Bot I, Pera T, Segers FM, Trompet S, van Gils JM, Roeten MK, Beckers CM, van Santbrink PJ, Janssen A, van Solingen C, Swildens J, de Boer HC, Peters EA, Bijkerk R, Rousch M, Doop M, Kuiper J, Schalij MJ, van der Wal AC, Richard S, van Berkel TJ, Pickering JG, Hiemstra PS, Goumans MJ, Rabelink TJ, de Vries AA, Quax PH, Jukema JW, Biessen EA, van Zonneveld AJ. van der Veer EP, et al. Among authors: swildens j. Circ Res. 2013 Oct 12;113(9):1065-75. doi: 10.1161/CIRCRESAHA.113.301302. Epub 2013 Aug 20. Circ Res. 2013. PMID: 23963726
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