A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation

por Böhm, Sybille, Splith, Victoria, Riedmayr, Lisa Maria, Rötzer, René Dominik, Gasparoni, Gilles, Nordström, Karl J. V., Wagner, Johanna Elisabeth, Hinrichsmeyer, Klara Sonnie, Walter, Jörn, Wahl-Schott, Christian, Fenske, Stefanie, Biel, Martin, Michalakis, Stylianos, Becirovic, Elvir
Publicado 2020

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

por Riedmayr, Lisa Maria, Hinrichsmeyer, Klara Sonnie, Thalhammer, Stefan Bernhard, Mittas, David Manuel, Karguth, Nina, Otify, Dina Yehia, Böhm, Sybille, Weber, Valentin Johannes, Bartoschek, Michael David, Splith, Victoria, Brümmer, Manuela, Ferreira, Raphael, Boon, Nanda, Wögenstein, Gabriele Maria, Grimm, Christian, Wijnholds, Jan, Mehlfeld, Verena, Michalakis, Stylianos, Fenske, Stefanie, Biel, Martin, Becirovic, Elvir
Publicado 2023