Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome

por Parrini, Martina, Naskar, Shovan, Alberti, Micol, Colombi, Ilaria, Morelli, Giovanni, Rocchi, Anna, Nanni, Marina, Piccardi, Federica, Charles, Severine, Ronzitti, Giuseppe, Mingozzi, Federico, Contestabile, Andrea, Cancedda, Laura
Publicado 2021

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

por Barbon, Elena, Kawecki, Charlotte, Marmier, Solenne, Sakkal, Aboud, Collaud, Fanny, Charles, Severine, Ronzitti, Giuseppe, Casari, Caterina, Christophe, Olivier D., Denis, Cécile V., Lenting, Peter J., Mingozzi, Federico
Publicado 2021

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

por Fitzpatrick, Zachary, Leborgne, Christian, Barbon, Elena, Masat, Elisa, Ronzitti, Giuseppe, van Wittenberghe, Laetitia, Vignaud, Alban, Collaud, Fanny, Charles, Séverine, Simon Sola, Marcelo, Jouen, Fabienne, Boyer, Olivier, Mingozzi, Federico
Publicado 2018

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

por Colella, Pasqualina, Sellier, Pauline, Costa Verdera, Helena, Puzzo, Francesco, van Wittenberghe, Laetitia, Guerchet, Nicolas, Daniele, Nathalie, Gjata, Bernard, Marmier, Solenne, Charles, Severine, Simon Sola, Marcelo, Ragone, Isabella, Leborgne, Christian, Collaud, Fanny, Mingozzi, Federico
Publicado 2018

A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia

por Zhukouskaya, Volha V., Jauze, Louisa, Charles, Séverine, Leborgne, Christian, Hilliquin, Stéphane, Sadoine, Jérémy, Slimani, Lotfi, Baroukh, Brigitte, van Wittenberghe, Laetitia, Danièle, Natalie, Rajas, Fabienne, Linglart, Agnès, Mingozzi, Federico, Chaussain, Catherine, Bardet, Claire, Ronzitti, Giuseppe
Publicado 2021

Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors

por Barbon, Elena, Ayme, Gabriel, Mohamadi, Amel, Ottavi, Jean‐François, Kawecki, Charlotte, Casari, Caterina, Verhenne, Sebastien, Marmier, Solenne, van Wittenberghe, Laetitia, Charles, Severine, Collaud, Fanny, Denis, Cecile V, Christophe, Olivier D, Mingozzi, Federico, Lenting, Peter J
Publicado 2020

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

por Ronzitti, Giuseppe, Bortolussi, Giulia, van Dijk, Remco, Collaud, Fanny, Charles, Severine, Leborgne, Christian, Vidal, Patrice, Martin, Samia, Gjata, Bernard, Sola, Marcelo Simon, van Wittenberghe, Laetitia, Vignaud, Alban, Veron, Philippe, Bosma, Piter J, Muro, Andres F, Mingozzi, Federico
Publicado 2016

Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector

por De Sabbata, Giulia, Boisgerault, Florence, Guarnaccia, Corrado, Iaconcig, Alessandra, Bortolussi, Giulia, Collaud, Fanny, Ronzitti, Giuseppe, Sola, Marcelo Simon, Vidal, Patrice, Rouillon, Jeremy, Charles, Severine, Nicastro, Emanuele, D’Antiga, Lorenzo, Ilyinskii, Petr, Mingozzi, Federico, Kishimoto, Takashi Kei, Muro, Andrés F.
Publicado 2020

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

por Cagin, Umut, Puzzo, Francesco, Gomez, Manuel Jose, Moya-Nilges, Maryse, Sellier, Pauline, Abad, Catalina, Van Wittenberghe, Laetitia, Daniele, Nathalie, Guerchet, Nicolas, Gjata, Bernard, Collaud, Fanny, Charles, Severine, Sola, Marcelo Simon, Boyer, Olivier, Krijnse-Locker, Jacomina, Ronzitti, Giuseppe, Colella, Pasqualina, Mingozzi, Federico
Publicado 2020

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

por Colella, Pasqualina, Sellier, Pauline, Gomez, Manuel J., Biferi, Maria G., Tanniou, Guillaume, Guerchet, Nicolas, Cohen-Tannoudji, Mathilde, Moya-Nilges, Maryse, van Wittenberghe, Laetitia, Daniele, Natalie, Gjata, Bernard, Krijnse-Locker, Jacomina, Collaud, Fanny, Simon-Sola, Marcelo, Charles, Severine, Cagin, Umut, Mingozzi, Federico
Publicado 2020

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

por Meliani, Amine, Boisgerault, Florence, Hardet, Romain, Marmier, Solenne, Collaud, Fanny, Ronzitti, Giuseppe, Leborgne, Christian, Costa Verdera, Helena, Simon Sola, Marcelo, Charles, Severine, Vignaud, Alban, van Wittenberghe, Laetitia, Manni, Giorgia, Christophe, Olivier, Fallarino, Francesca, Roy, Christopher, Michaud, Alicia, Ilyinskii, Petr, Kishimoto, Takashi Kei, Mingozzi, Federico
Publicado 2018

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

por Collaud, Fanny, Bortolussi, Giulia, Guianvarc’h, Laurence, Aronson, Sem J., Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine G., Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia v., Martin, Samia, Le Bec, Christine, Bosma, Piter J., Muro, Andres F., Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico
Publicado 2018

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates

por Costa-Verdera, Helena, Collaud, Fanny, Riling, Christopher R., Sellier, Pauline, Nordin, Jayme M. L., Preston, G. Michael, Cagin, Umut, Fabregue, Julien, Barral, Simon, Moya-Nilges, Maryse, Krijnse-Locker, Jacomina, van Wittenberghe, Laetitia, Daniele, Natalie, Gjata, Bernard, Cosette, Jeremie, Abad, Catalina, Simon-Sola, Marcelo, Charles, Severine, Li, Mathew, Crosariol, Marco, Antrilli, Tom, Quinn, William J., Gross, David A., Boyer, Olivier, Anguela, Xavier M., Armour, Sean M., Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico
Publicado 2021