Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy by Nicole Armbruster, Annalisa Lattanzi, Matthieu Jeavons, Laetitia Van Wittenberghe, Bernard Gjata, Thibaut Marais, Samia Martin, Alban Vignaud, Thomas Voit, Fulvio Mavilio, Martine Barkats, Ana Buj-Bello

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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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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome by Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, Fanny Collaud, Severine Charles, Christian Leborgne, Patrice Vidal, Samia Martin, Bernard Gjata, Marcelo Simon Sola, Laetitia van Wittenberghe, Alban Vignaud, Philippe Veron, Piter J Bosma, Andres F Muro, Federico Mingozzi

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Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors by Amine Meliani, Florence Boisgerault, Zachary Fitzpatrick, Solenne Marmier, Christian Leborgne, Fanny Collaud, Marcelo Simon Sola, Severine Charles, Giuseppe Ronzitti, Alban Vignaud, Laetitia van Wittenberghe, Beatrice Marolleau, Fabienne Jouen, Sisareuth Tan, Olivier Boyer, Olivier Christophe, Alain R. Brisson, Casey A. Maguire, Federico Mingozzi

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