| Title | Authors | Publication | Year |
|---|
| PCDH15 dual-AAV gene therapy for deafness and blindness in Usher syndrome type 1F models | Maryna V Ivanchenko, Daniel M Hathaway, Eric M Mulhall, Kevin Ta Booth, Mantian Wang, Cole W Peters, Alex J Klein, Xinlan Chen, Yaqiao Li, Bence György, David P Corey | J Clin Invest. | 2024 |
| High-efficiency base editing in the retina in primates and human tissues | Alissa Muller, Jack Sullivan, Wibke Schwarzer, Mantian Wang, Cindy Park-Windhol, Pascal W Hasler, Lucas Janeschitz-Kriegl, Mert Duman, Beryll Klingler, Jane Matsell, Simon Manuel Hostettler, Patricia Galliker, Yanyan Hou, Pierre Balmer, Tamás Virág, Luis Alberto Barrera, Lauren Young, Quan Xu, Dániel Péter Magda, Ferenc Kilin, Arogya Khadka, Pierre-Henri Moreau, Lyne Fellmann, Thierry Azoulay, Mathieu Quinodoz, Duygu Karademir, Juna Leppert, Alex Fratzl, Georg Kosche, Ruchi Sharma, Jair Montford, Marco Cattaneo, Mikaël Croyal, Therese Cronin, Simone Picelli, Alice Grison, Cameron S Cowan, Ákos Kusnyerik, Philipp Anders, Magdalena Renner, Zoltán Zsolt Nagy, Arnold Szabó, Kapil Bharti, Carlo Rivolta, Hendrik P N Scholl, David Bryson, Giuseppe Ciaramella, Botond Roska, Bence György | Nature Medicine | 2025 |
| Detection of elusive DNA copy-number variations in hereditary disease and cancer through the use of noncoding and off-target sequencing reads | Mathieu Quinodoz, Karolina Kaminska, Francesca Cancellieri, Ji Hoon Han, Virginie G Peter, Elifnaz Celik, Lucas Janeschitz-Kriegl, Nils Schärer, Daniela Hauenstein, Bence György, Giacomo Calzetti, Vincent Hahaut, Sónia Custódio, Ana Cristina Sousa, Yuko Wada, Yusuke Murakami, Almudena Avila Fernández, Cristina Rodilla Hernández, Pablo Minguez, Carmen Ayuso, Koji M Nishiguchi, Cristina Santos, Luisa Coutinho Santos, Viet H Tran, Veronika Vaclavik, Hendrik P N Scholl, Carlo Rivolta | Am J Hum Genet. | 2024 |
| c.-61G>A in OVOL2 is a Pathogenic 5′ Untranslated Region Variant Causing Posterior Polymorphous Corneal Dystrophy 1 | Lucas Janeschitz-Kriegl, Dhryata Kamdar, Mathieu Quinodoz, Karolina Kaminska, Marc Folcher, Bence György, Peter Meyer, Andreas Wild, Pascal Escher, Hendrik P N Scholl, Carlo Rivolta, David Goldblum | Cornea | 2021 |
| Rare occult macular dystrophy with a pathogenic variant in the RP1L1 gene in a patient of Swiss descent | Olga Zabek, Ioannis Lamprakis, Annekatrin Rickmann, Giacomo Calzetti, Bence György, Hendrik P N Scholl, Maria Della Volpe Waizel | American journal of ophthalmology case reports | 2022 |
| CRISPR-Cas9 treatment partially restores amyloid-β 42/40 in human fibroblasts with the Alzheimer’s disease PSEN 1 M146L mutation | Evangelos Konstantinidis, Agnieszka Molisak, Florian Perrin, Linn Streubel-Gallasch, Sarah Fayad, Daniel Y Kim, Karl Petri, Martin J Aryee, Ximena Aguilar, Bence György, Vilmantas Giedraitis, J Keith Joung, Vikram Pattanayak, Magnus Essand, Anna Erlandsson, Oksana Berezovska, Martin Ingelsson | Molecular therapy. Nucleic acids | 2022 |
| Differentially Expressed Inflammation-Regulating MicroRNAs in Oligoarticular Juvenile Idiopathic Arthritis | Sarah M McAlpine, Sarah E Roberts, Breanna K V Hargreaves, Claire Bullock, Suzanne Ramsey, Elizabeth Stringer, Bianca Lang, Adam Huber, Bence György, Fruzsina Erdélyi, Thomas B Issekutz, Beáta Dérfalvi | The Journal of rheumatology | 2022 |
| Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss | Bence György, Carl Nist-Lund, Bifeng Pan, Yukako Asai, K Domenica Karavitaki, Benjamin P Kleinstiver, Sara P Garcia, Mikołaj P Zaborowski, Paola Solanes, Sofia Spataro, Bernard L Schneider, J Keith Joung, Gwenaëlle S G Géléoc, Jeffrey R Holt, David P Corey | Nature medicine | 2019 |
| High levels of AAV vector integration into CRISPR-induced DNA breaks | Killian S Hanlon, Benjamin P Kleinstiver, Sara P Garcia, Mikołaj P Zaborowski, Adrienn Volak, Stefan E Spirig, Alissa Muller, Alexander A Sousa, Shengdar Q Tsai, Niclas E Bengtsson, Camilla Lööv, Martin Ingelsson, Jeffrey S Chamberlain, David P Corey, Martin J Aryee, J Keith Joung, Xandra O Breakefield, Casey A Maguire, Bence György | Nature communications | 2019 |
| Membrane-bound Gaussia luciferase as a tool to track shedding of membrane proteins from the surface of extracellular vesicles | Mikołaj Piotr Zaborowski, Pike See Cheah, Xuan Zhang, Isabella Bushko, Kyungheon Lee, Alessandro Sammarco, Valentina Zappulli, Sybren Lein Nikola Maas, Ryan M Allen, Purva Rumde, Bence György, Massimo Aufiero, Markus W Schweiger, Charles Pin-Kuang Lai, Ralph Weissleder, Hakho Lee, Kasey C Vickers, Bakhos A Tannous, Xandra O Breakefield | Scientific reports | 2019 |
| Genome and base editing for genetic hearing loss | Philipp Niggemann, Bence György, Zheng-Yi Chen | Hearing research | 2020 |
| Mutant Allele-Specific CRISPR Disruption in DYT1 Dystonia Fibroblasts Restores Cell Function | Lilian Cruz, Bence György, Pike See Cheah, Benjamin P Kleinstiver, William A Eimer, Sara P Garcia, Nutan Sharma, Laurie J Ozelius, D Cristopher Bragg, J Keith Joung, Osmar Norberto de Souza, Luis Fernando Saraiva Macedo Timmers, Xandra O Breakefield | Molecular therapy. Nucleic acids | 2020 |
| Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin | Pike-See Cheah, Shilpa Prabhakar, David Yellen, Roberta L Beauchamp, Xuan Zhang, Shingo Kasamatsu, Roderick T Bronson, Elizabeth A Thiele, David J Kwiatkowski, Anat Stemmer-Rachamimov, Bence György, King-Hwa Ling, Masao Kaneki, Bakhos A Tannous, Vijaya Ramesh, Casey A Maguire, Xandra O Breakefield | Science advances | 2021 |
| Effect of prolactin on normal and keratoconus human corneal stromal fibroblasts in vitro | Philipp Anders, Xuefei Song, Bence György, Nora Szentmary, Berthold Seitz, Zisis Gatzioufas | PloS one | 2021 |