Improving Translation by Identifying Evidence for More Human-Relevant Preclinical Strategies by Merel Ritskes-Hoitinga, Cathalijn Leenaars, Wouter Beumer, Tineke Coenen-de Roo, Frans Stafleu, Franck L. B. Meijboom

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The gene expression profile of CD11c+ CD8α- dendritic cells in the pre-diabetic pancreas of the NOD mouse. by Wouter Beumer, Jojanneke M C Welzen-Coppens, Cornelia G van Helden-Meeuwsen, Sinead M Gibney, Hemmo A Drexhage, Marjan A Versnel

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Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C by Melita Kaltak, Petra de Bruijn, Davide Piccolo, Sang-Eun Lee, Kalyan Dulla, Thomas Hoogenboezem, Wouter Beumer, Andrew R. Webster, Rob W.J. Collin, Michael E. Cheetham, Gerard Platenburg, Jim Swildens

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

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A pathogenic proteolysis–resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function by Hyeongju Kim, Sophie Lenoir, Angela Helfricht, Taeyang Jung, Zhana K. Karneva, Yejin Lee, Wouter Beumer, Geert B. van der Horst, Herma Anthonijsz, Levi C.M. Buil, Frits van der Ham, Gerard J. Platenburg, Pasi Purhonen, Hans Hebert, Sandrine Humbert, Frédéric Saudou, Pontus Klein, Ji-Joon Song

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