In vivo liver targeted genome editing as therapeutic approach: progresses and challenges by Chiara Simoni, Chiara Simoni, Elena Barbon, Andrés F. Muro, Alessio Cantore, Alessio Cantore

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Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice by Michela Lisjak, Alessia De Caneva, Thibaut Marais, Elena Barbon, Elena Barbon, Maria Grazia Biferi, Fabiola Porro, Adi Barzel, Lorena Zentilin, Mark A. Kay, Federico Mingozzi, Federico Mingozzi, Federico Mingozzi, Andrés F. Muro

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Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics by Elena Barbon, Mattia Ferrarese, Laetitia van Wittenberghe, Peggy Sanatine, Giuseppe Ronzitti, Fanny Collaud, Pasqualina Colella, Mirko Pinotti, Federico Mingozzi

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Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System by Annalisa Lattanzi, Stephanie Duguez, Arianna Moiani, Araksya Izmiryan, Elena Barbon, Samia Martin, Kamel Mamchaoui, Vincent Mouly, Francesco Bernardi, Fulvio Mavilio, Matteo Bovolenta

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Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction by Zachary Fitzpatrick, Christian Leborgne, Elena Barbon, Elisa Masat, Giuseppe Ronzitti, Laetitia van Wittenberghe, Alban Vignaud, Fanny Collaud, Séverine Charles, Marcelo Simon Sola, Fabienne Jouen, Olivier Boyer, Federico Mingozzi

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Pathological modeling of glycogen storage disease type III with CRISPR/Cas9 edited human pluripotent stem cells by Lucille Rossiaud, Lucille Rossiaud, Lucille Rossiaud, Lucille Rossiaud, Lucille Rossiaud, Pascal Fragner, Pascal Fragner, Pascal Fragner, Elena Barbon, Elena Barbon, Antoine Gardin, Antoine Gardin, Manon Benabides, Manon Benabides, Manon Benabides, Emilie Pellier, Emilie Pellier, Emilie Pellier, Jérémie Cosette, Lina El Kassar, Lina El Kassar, Lina El Kassar, Karine Giraud-Triboult, Karine Giraud-Triboult, Karine Giraud-Triboult, Xavier Nissan, Xavier Nissan, Xavier Nissan, Giuseppe Ronzitti, Giuseppe Ronzitti, Lucile Hoch, Lucile Hoch, Lucile Hoch

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Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors by Elena Barbon, Gabriel Ayme, Amel Mohamadi, Jean‐François Ottavi, Charlotte Kawecki, Caterina Casari, Sebastien Verhenne, Solenne Marmier, Laetitia van Wittenberghe, Severine Charles, Fanny Collaud, Cecile V Denis, Olivier D Christophe, Federico Mingozzi, Peter J Lenting

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Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy by Sergio Muñoz, Joan Bertolin, Veronica Jimenez, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Laia Vilà, Victor Sacristan, Elena Barbon, Giuseppe Ronzitti, Jihad El Andari, Warut Tulalamba, Quang Hong Pham, Jesus Ruberte, Thierry VandenDriessche, Marinee K. Chuah, Dirk Grimm, Federico Mingozzi, Fatima Bosch

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Illustrated State‐of‐the‐Art Capsules of the ISTH 2022 Congress by Robert A. Ariëns, Beverley J. Hunt, Ejaife O. Agbani, Josefin Ahnström, Robert Ahrends, Raza Alikhan, Alice Assinger, Zsuzsa Bagoly, Alessandra Balduini, Elena Barbon, Christopher D. Barrett, Paul Batty, Jorge David Aivazoglou Carneiro, Wee Shian Chan, Moniek deMaat, Kerstin deWit, Cécile Denis, Martin H. Ellis, Renee Eslick, Hongxia Fu, Catherine P. M. Hayward, Benoit Ho‐Tin‐Noé, Frederikus A. Klok, Riten Kumar, Karin Leiderman, Rustem I. Litvinov, Nigel Mackman, Zoe McQuilten, Matthew D. Neal, William A. E. Parker, Roger J. S. Preston, Julie Rayes, Alireza R. Rezaie, Lara N. Roberts, Bianca Rocca, Susan Shapiro, Deborah M. Siegal, Lirlândia P. Sousa, Katsue Suzuki‐Inoue, Tahira Zafar, Jiaxi Zhou

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