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Page 1
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies.
Leborgne C, Barbon E, Alexander JM, Hanby H, Delignat S, Cohen DM, Collaud F, Muraleetharan S, Lupo D, Silverberg J, Huang K, van Wittengerghe L, Marolleau B, Miranda A, Fabiano A, Daventure V, Beck H, Anguela XM, Ronzitti G, Armour SM, Lacroix-Desmazes S, Mingozzi F. Leborgne C, et al. Among authors: barbon e. Nat Med. 2020 Jul;26(7):1096-1101. doi: 10.1038/s41591-020-0911-7. Epub 2020 Jun 1. Nat Med. 2020. PMID: 32483358 Free article.
Illustrated State-of-the-Art Capsules of the ISTH 2022 Congress.
Ariëns RA, Hunt BJ, Agbani EO, Ahnström J, Ahrends R, Alikhan R, Assinger A, Bagoly Z, Balduini A, Barbon E, Barrett CD, Batty P, Carneiro JDA, Chan WS, de Maat M, de Wit K, Denis C, Ellis MH, Eslick R, Fu H, Hayward CPM, Ho-Tin-Noé B, Klok FA, Kumar R, Leiderman K, Litvinov RI, Mackman N, McQuilten Z, Neal MD, Parker WAE, Preston RJS, Rayes J, Rezaie AR, Roberts LN, Rocca B, Shapiro S, Siegal DM, Sousa LP, Suzuki-Inoue K, Zafar T, Zhou J. Ariëns RA, et al. Among authors: barbon e. Res Pract Thromb Haemost. 2022 Jul 6;6(5):e12747. doi: 10.1002/rth2.12747. eCollection 2022 Jul. Res Pract Thromb Haemost. 2022. PMID: 35814801 Free PMC article. Review.
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.
Muñoz S, Bertolin J, Jimenez V, Jaén ML, Garcia M, Pujol A, Vilà L, Sacristan V, Barbon E, Ronzitti G, El Andari J, Tulalamba W, Pham QH, Ruberte J, VandenDriessche T, Chuah MK, Grimm D, Mingozzi F, Bosch F. Muñoz S, et al. Among authors: barbon e. Mol Metab. 2024 Mar;81:101899. doi: 10.1016/j.molmet.2024.101899. Epub 2024 Feb 10. Mol Metab. 2024. PMID: 38346589 Free PMC article.
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.
Fitzpatrick Z, Leborgne C, Barbon E, Masat E, Ronzitti G, van Wittenberghe L, Vignaud A, Collaud F, Charles S, Simon Sola M, Jouen F, Boyer O, Mingozzi F. Fitzpatrick Z, et al. Among authors: barbon e. Mol Ther Methods Clin Dev. 2018 Feb 13;9:119-129. doi: 10.1016/j.omtm.2018.02.003. eCollection 2018 Jun 15. Mol Ther Methods Clin Dev. 2018. PMID: 29766022 Free PMC article.
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.
Lattanzi A, Duguez S, Moiani A, Izmiryan A, Barbon E, Martin S, Mamchaoui K, Mouly V, Bernardi F, Mavilio F, Bovolenta M. Lattanzi A, et al. Among authors: barbon e. Mol Ther Nucleic Acids. 2017 Jun 16;7:11-19. doi: 10.1016/j.omtn.2017.02.004. Epub 2017 Feb 10. Mol Ther Nucleic Acids. 2017. PMID: 28624187 Free PMC article.
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