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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: ritsema t. PLoS One. 2019 Jun 28;14(6):e0219182. doi: 10.1371/journal.pone.0219182. eCollection 2019. PLoS One. 2019. PMID: 31251792 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: ritsema t. 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.
AAV-mediated in vivo knockdown of luciferase using combinatorial RNAi and U1i.
Koornneef A, van Logtenstein R, Timmermans E, Pisas L, Blits B, Abad X, Fortes P, Petry H, Konstantinova P, Ritsema T. Koornneef A, et al. Among authors: ritsema t. Gene Ther. 2011 Sep;18(9):929-35. doi: 10.1038/gt.2011.41. Epub 2011 Apr 7. Gene Ther. 2011. PMID: 21472008 Free PMC article.
68 results