Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290

por Collin, Rob WJ, den Hollander, Anneke I, van der Velde-Visser, Saskia D, Bennicelli, Jeannette, Bennett, Jean, Cremers, Frans PM
Publicado 2012

Impaired ABCA1/ABCG1-mediated lipid efflux in the mouse retinal pigment epithelium (RPE) leads to retinal degeneration

por Storti, Federica, Klee, Katrin, Todorova, Vyara, Steiner, Regula, Othman, Alaa, van der Velde-Visser, Saskia, Samardzija, Marijana, Meneau, Isabelle, Barben, Maya, Karademir, Duygu, Pauzuolyte, Valda, Boye, Sanford L, Blaser, Frank, Ullmer, Christoph, Dunaief, Joshua L, Hornemann, Thorsten, Rohrer, Lucia, den Hollander, Anneke, von Eckardstein, Arnold, Fingerle, Jürgen, Maugeais, Cyrille, Grimm, Christian
Publicado 2019