| IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies C Leborgne, E Barbon, JM Alexander, H Hanby, S Delignat, DM Cohen, ... Nature medicine 26 (7), 1096-1101, 2020 | 262 | 2020 |
| Influence of pre-existing anti-capsid neutralizing and binding antibodies on AAV vector transduction Z Fitzpatrick, C Leborgne, E Barbon, E Masat, G Ronzitti, ... Molecular therapy Methods & clinical development 9, 119-129, 2018 | 177 | 2018 |
| Correction of the exon 2 duplication in DMD myoblasts by a single CRISPR/Cas9 system A Lattanzi, S Duguez, A Moiani, A Izmiryan, E Barbon, S Martin, ... Molecular Therapy-Nucleic Acids 7, 11-19, 2017 | 67 | 2017 |
| Molecular basis and therapeutic strategies to rescue factor IX variants that affect splicing and protein function M Tajnik, ME Rogalska, E Bussani, E Barbon, D Balestra, M Pinotti, ... PLoS Genetics 12 (5), e1006082, 2016 | 44 | 2016 |
| Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model I Donadon, M Pinotti, K Rajkowska, G Pianigiani, E Barbon, E Morini, ... Human molecular genetics 27 (14), 2466-2476, 2018 | 43 | 2018 |
| Regulation of a strong F9 cryptic 5′ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides D Balestra, E Barbon, D Scalet, N Cavallari, D Perrone, S Zanibellato, ... Human molecular genetics 24 (17), 4809-4816, 2015 | 35 | 2015 |
| Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor E Barbon, C Kawecki, S Marmier, A Sakkal, F Collaud, S Charles, ... Gene Therapy 30 (3), 245-254, 2023 | 24 | 2023 |
| Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors E Barbon, G Ayme, A Mohamadi, JF Ottavi, C Kawecki, C Casari, ... EMBO molecular medicine 12 (4), e11298, 2020 | 23 | 2020 |
| An engineered tale-transcription factor rescues transcription of factor VII impaired by promoter mutations and enhances its endogenous expression in hepatocytes E Barbon, S Pignani, A Branchini, F Bernardi, M Pinotti, M Bovolenta Scientific reports 6 (1), 28304, 2016 | 19 | 2016 |
| Promoterless gene targeting approach combined to CRISPR/Cas9 efficiently corrects hemophilia B phenotype in neonatal mice M Lisjak, A De Caneva, T Marais, E Barbon, MG Biferi, F Porro, A Barzel, ... Frontiers in Genome Editing 4, 785698, 2022 | 16 | 2022 |
| Recombinant adeno-associated viral vectors expressing human coagulation FIX-E456H variant in hemophilia B mice S Le Quellec, AP Dane, E Barbon, JC Bordet, F Mingozzi, Y Dargaud, ... Thrombosis and Haemostasis 119 (12), 1956-1967, 2019 | 11 | 2019 |
| Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy S Muñoz, J Bertolin, V Jimenez, ML Jaén, M Garcia, A Pujol, L Vilà, ... Molecular Metabolism 81, 101899, 2024 | 9 | 2024 |
| Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts S Pignani, F Zappaterra, E Barbon, A Follenzi, M Bovolenta, F Bernardi, ... Biochimica et Biophysica Acta (BBA)-Gene Regulatory Mechanisms 1862 (6), 619-624, 2019 | 8 | 2019 |
| Pathological modeling of glycogen storage disease type III with CRISPR/Cas9 edited human pluripotent stem cells L Rossiaud, P Fragner, E Barbon, A Gardin, M Benabides, E Pellier, ... Frontiers in Cell and Developmental Biology 11, 1163427, 2023 | 4 | 2023 |
| Illustrated State‐of‐the‐Art Capsules of the ISTH 2022 Congress RA Ariëns, BJ Hunt, EO Agbani, J Ahnström, R Ahrends, R Alikhan, ... Research and Practice in Thrombosis and Haemostasis 6 (5), e12747, 2022 | 4 | 2022 |
| Transposon-mediated generation of cellular and mouse models of splicing mutations to assess the efficacy of snRNA-based therapeutics E Barbon, M Ferrarese, L Van Wittenberghe, P Sanatine, G Ronzitti, ... Molecular Therapy-Nucleic Acids 5, 2016 | 2 | 2016 |
| Influence of pre-existing anti-capsid neutralizing and binding antibodies on AAV-mediated liver transduction Z Fitzpatrick, C Leborgne, E Barbon, E Masat, L Van Wittenberg, ... MOLECULAR THERAPY 25 (5), 182-182, 2017 | 1 | 2017 |
| In vivo liver targeted genome editing as therapeutic approach: progresses and challenges C Simoni, E Barbon, AF Muro, A Cantore Frontiers in Genome Editing 6, 1458037, 2024 | | 2024 |
| Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model (vol 27, pg 2466, 2018) I Donadon, M Pinotti, K Rajkowska, G Pianigiani, E Barbon, E Morini, ... HUMAN MOLECULAR GENETICS, 2024 | | 2024 |
| Liver fibrosis negatively impacts in vivo gene transfer to hepatocytes A Cantore, C Simoni, J Nozi, F Starinieri, T La Bella, E Manta, C Negri, ... | | 2024 |
