AAV Vectors for FRET-Based Analysis of Protein-Protein Interactions in Photoreceptor Outer Segments

por Becirovic, Elvir, Böhm, Sybille, Nguyen, Ong N. P., Riedmayr, Lisa M., Hammelmann, Verena, Schön, Christian, Butz, Elisabeth S., Wahl-Schott, Christian, Biel, Martin, Michalakis, Stylianos
Publicado 2016

Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation

por Panagiotopoulos, Anna-Lena, Karguth, Nina, Pavlou, Marina, Böhm, Sybille, Gasparoni, Gilles, Walter, Jörn, Graf, Alexander, Blum, Helmut, Biel, Martin, Riedmayr, Lisa Maria, Becirovic, Elvir
Publicado 2020

Peripherin-2 and Rom-1 have opposing effects on rod outer segment targeting of retinitis pigmentosa-linked peripherin-2 mutants

por Böhm, Sybille, Riedmayr, Lisa M., Nguyen, O. N. Phuong, Gießl, Andreas, Liebscher, Toni, Butz, Elisabeth S., Schön, Christian, Michalakis, Stylianos, Wahl-Schott, Christian, Biel, Martin, Becirovic, Elvir
Publicado 2017

In Vivo Analysis of Disease-Associated Point Mutations Unveils Profound Differences in mRNA Splicing of Peripherin-2 in Rod and Cone Photoreceptors

por Becirovic, Elvir, Böhm, Sybille, Nguyen, Ong Nam Phuong, Riedmayr, Lisa Maria, Koch, Mirja Annika, Schulze, Elisabeth, Kohl, Susanne, Borsch, Oliver, Santos-Ferreira, Tiago, Ader, Marius, Michalakis, Stylianos, Biel, Martin
Publicado 2016

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