A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation S Böhm, V Splith, LM Riedmayr, RD Rötzer, G Gasparoni, KJV Nordström, ... Science advances 6 (34), eaba5614, 2020 | 44 | 2020 |
A deep intronic CLRN1 (USH3A) founder mutation generates an aberrant exon and underlies severe Usher syndrome on the Arabian Peninsula AO Khan, E Becirovic, C Betz, C Neuhaus, J Altmüller, L Maria Riedmayr, ... Scientific reports 7 (1), 1411, 2017 | 44 | 2017 |
AAV vectors for FRET-based analysis of protein-protein interactions in photoreceptor outer segments E Becirovic, S Böhm, ONP Nguyen, LM Riedmayr, V Hammelmann, ... Frontiers in neuroscience 10, 356, 2016 | 28 | 2016 |
In Vivo Analysis of Disease-Associated Point Mutations Unveils Profound Differences in mRNA Splicing of Peripherin-2 in Rod and Cone Photoreceptors E Becirovic, S Böhm, ONP Nguyen, LM Riedmayr, MA Koch, E Schulze, ... PLoS genetics 12 (1), e1005811, 2016 | 26 | 2016 |
Antisense oligonucleotide-and CRISPR-Cas9-mediated rescue of mRNA splicing for a deep intronic CLRN1 mutation AL Panagiotopoulos, N Karguth, M Pavlou, S Böhm, G Gasparoni, ... Molecular Therapy-Nucleic Acids 21, 1050-1061, 2020 | 15 | 2020 |
Peripherin-2 and Rom-1 have opposing effects on rod outer segment targeting of retinitis pigmentosa-linked peripherin-2 mutants S Böhm, LM Riedmayr, ONP Nguyen, A Gießl, T Liebscher, ES Butz, ... Scientific reports 7 (1), 2321, 2017 | 15 | 2017 |
dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy LM Riedmayr, KS Hinrichsmeyer, N Karguth, S Böhm, V Splith, ... Nature Protocols 17 (3), 781-818, 2022 | 11 | 2022 |
Construction and cloning of minigenes for in vivo analysis of potential splice mutations LM Riedmayr, S Böhm, S Michalakis, E Becirovic Bio-protocol 8 (5), e2760-e2760, 2018 | 7 | 2018 |
mRNA trans-splicing dual AAV vectors for (epi) genome editing and gene therapy LM Riedmayr, KS Hinrichsmeyer, SB Thalhammer, DM Mittas, N Karguth, ... Nature Communications 14 (1), 6578, 2023 | 6 | 2023 |
Enigmatic rhodopsin mutation creates an exceptionally strong splice acceptor site LM Riedmayr, S Böhm, M Biel, E Becirovic Human Molecular Genetics 29 (2), 295-304, 2020 | 6 | 2020 |
SMaRT for therapeutic purposes LM Riedmayr Chimeric RNA: Methods and Protocols, 219-232, 2020 | 5 | 2020 |
Randomizing the human genome by engineering recombination between repeat elements J Koeppel, R Ferreira, T Vanderstichele, LM Riedmayr, EM Peets, ... bioRxiv, 2024.01. 22.576745, 2024 | 3 | 2024 |
Machine Learning and Directed Evolution of Base Editing Enzymes RM Perrotta, S Vinke, R Ferreira, M Moret, A Mahas, A Chiappino-Pepe, ... bioRxiv, 2024.05. 17.594556, 2024 | | 2024 |