2024
A base editor for correcting deafness (Review)
Nature Biomedical Engineering
Detection of elusive DNA copy-number variations in hereditary disease and cancer through the use of noncoding and off-target sequencing reads
American Journal of Human Genetics
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
2023
PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F
bioRxiv [preprint]
2022
CRISPR-Cas9 treatment partially restores amyloid-b 42/40 in human fibroblasts with the Alzheimer’s disease PSEN1 M146L mutation
Molecular Therapy - Nucleic Acids
Rare occult macular dystrophy with a pathogenic variant in the RP1L1 gene in a patient of Swiss descent
American Journal of Ophthalmology Case Reports
c.-61G>A in OVOL2 is a Pathogenic 5' Untranslated Region Variant Causing Posterior Polymorphous Corneal Dystrophy 1
Cornea
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
2020
Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin
Science Advances
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
Mutant Allele-Specific CRISPR Disruption in DYT1 Dystonia Fibroblasts Restores Cell Function
Molecular Therapy Nucleic Acids
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
Genome and base editing for genetic hearing loss
Hearing Research
Philipp Niggemann, Bence György, Zheng-Yi Chen
2019
Membrane-bound Gaussia luciferase as a tool to track shedding of membrane proteins from the surface of extracellular vesicles
Scientific Reports
MikoÅ‚aj 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
High levels of AAV vector integration into CRISPR-induced DNA breaks
Nature Communications
Killian S Hanlon, Benjamin P Kleinstiver, Sara P Garcia, MikoÅ‚aj 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
Allele-specific gene editing prevents deafness in a
model of dominant progressive hearing loss
Nature Medicine
Bence György, Carl Nist-Lund, Bifeng Pan, Yukako Asai, K Domenica Karavitaki, Benjamin P Kleinstiver, Sara P Garcia, MikoÅ‚aj P Zaborowski, Paola Solanes, Sofia Spataro, Bernard L Schneider, J Keith Joung, Gwenaëlle S G Géléoc, Jeffrey R Holt, David P Corey
older (key papers only)
TMC1 Forms the Pore of Mechanosensory Transduction Channels in Vertebrate Inner Ear Hair Cells
Neuron
Bifeng Pan, Nurunisa Akyuz, Xiao-Ping Liu, Yukako Asai, Carl Nist-Lund, Kiyoto Kurima, Bruce H Derfler, Bence György, Walrati Limapichat, Sanket Walujkar, Lahiru N Wimalasena, Marcos Sotomayor, David P Corey, Jeffrey R Holt
Alzheimer's Disease-Associated β-Amyloid Is Rapidly Seeded by Herpesviridae to Protect against Brain Infection
Neuron
William A Eimer, Deepak Kumar Vijaya Kumar, Nanda Kumar Navalpur Shanmugam, Alex S Rodriguez, Teryn Mitchell, Kevin J Washicosky, Bence György, Xandra O Breakefield, Rudolph E Tanzi, Robert D Moir
Rescue of Hearing by Gene Delivery to Inner-Ear Hair
Cells Using Exosome-Associated AAV
Molecular Therapy
Bence György, Cyrille Sage, Artur A Indzhykulian, Deborah I Scheffer, Alain R Brisson, Sisareuth Tan, Xudong Wu, Adrienn Volak, Dakai Mu, Panos I Tamvakologos, Yaqiao Li, Zachary Fitzpatrick, Maria Ericsson, Xandra O Breakefield, David P Corey, Casey A Maguire
Emerging role of extracellular vesicles in inflammatory diseases
Nature Reviews Rheumatology
Edit I Buzas, Bence György, György Nagy, András Falus, Steffen Gay
Detection and isolation of cell-derived microparticles
are compromised by protein complexes resulting from
shared biophysical parameters
Blood
Bence György, Károly Módos, Eva Pállinger, Krisztina Pálóczi, Mária Pásztói, Petra Misják, Mária A Deli, Aron Sipos, Anikó Szalai, István Voszka, Anna Polgár, Kálmán Tóth, Mária Csete, György Nagy, Steffen Gay, András Falus, Agnes Kittel, Edit I Buzás