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

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