Improving Translation by Identifying Evidence for More Human-Relevant Preclinical Strategies

por Ritskes-Hoitinga, Merel, Leenaars, Cathalijn, Beumer, Wouter, Coenen-de Roo, Tineke, Stafleu, Frans, Meijboom, Franck L. B.
Publicado 2020

The Gene Expression Profile of CD11c(+)CD8α(−) Dendritic Cells in the Pre-Diabetic Pancreas of the NOD Mouse

por Beumer, Wouter, Welzen-Coppens, Jojanneke M. C., van Helden-Meeuwsen, Cornelia G., Gibney, Sinead M., Drexhage, Hemmo A., Versnel, Marjan A.
Publicado 2014

Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C

por Kaltak, Melita, de Bruijn, Petra, Piccolo, Davide, Lee, Sang-Eun, Dulla, Kalyan, Hoogenboezem, Thomas, Beumer, Wouter, Webster, Andrew R., Collin, Rob W.J., Cheetham, Michael E., Platenburg, Gerard, Swildens, Jim
Publicado 2023

Prevalence of interferon type I signature in CD14 monocytes of patients with Sjögren's syndrome and association with disease activity and BAFF gene expression

por Brkic, Zana, Maria, Naomi I, van Helden-Meeuwsen, Cornelia G, van de Merwe, Joop P, van Daele, Paul L, Dalm, Virgil A, Wildenberg, Manon E, Beumer, Wouter, Drexhage, Hemmo A, Versnel, Marjan A
Publicado 2013

Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis

por Beumer, Wouter, Swildens, Jim, Leal, Teresinha, Noel, Sabrina, Anthonijsz, Herma, van der Horst, Geert, Kuiperij-Boersma, Hester, Potman, Marko, van Putten, Charlotte, Biasutto, Patricia, Platenburg, Gerard, de Jonge, Hugo, Henig, Noreen, Ritsema, Tita
Publicado 2019

Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

por Dulla, Kalyan, Aguila, Monica, Lane, Amelia, Jovanovic, Katarina, Parfitt, David A., Schulkens, Iris, Chan, Hee Lam, Schmidt, Iris, Beumer, Wouter, Vorthoren, Lars, Collin, Rob W.J., Garanto, Alejandro, Duijkers, Lonneke, Brugulat-Panes, Anna, Semo, Ma’ayan, Vugler, Anthony A., Biasutto, Patricia, Adamson, Peter, Cheetham, Michael E.
Publicado 2018

A pathogenic proteolysis–resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function

por Kim, Hyeongju, Lenoir, Sophie, Helfricht, Angela, Jung, Taeyang, Karneva, Zhana K., Lee, Yejin, Beumer, Wouter, van der Horst, Geert B., Anthonijsz, Herma, Buil, Levi C.M., van der Ham, Frits, Platenburg, Gerard J., Purhonen, Pasi, Hebert, Hans, Humbert, Sandrine, Saudou, Frédéric, Klein, Pontus, Song, Ji-Joon
Publicado 2022

Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations

por Dulla, Kalyan, Slijkerman, Ralph, van Diepen, Hester C., Albert, Silvia, Dona, Margo, Beumer, Wouter, Turunen, Janne J., Chan, Hee Lam, Schulkens, Iris A., Vorthoren, Lars, den Besten, Cathaline, Buil, Levi, Schmidt, Iris, Miao, Jiayi, Venselaar, Hanka, Zang, Jingjing, Neuhauss, Stephan C.F., Peters, Theo, Broekman, Sanne, Pennings, Ronald, Kremer, Hannie, Platenburg, Gerard, Adamson, Peter, de Vrieze, Erik, van Wijk, Erwin
Publicado 2021