Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
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
Published in Nature communications (05.10.2018)
Published in Nature communications (05.10.2018)
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Journal Article
Exposure to wild-type AAV drives distinct capsid immunity profiles in humans
Kuranda, Klaudia, Jean-Alphonse, Priscilla, Leborgne, Christian, Hardet, Romain, Collaud, Fanny, Marmier, Solenne, Costa Verdera, Helena, Ronzitti, Giuseppe, Veron, Philippe, Mingozzi, Federico
Published in The Journal of clinical investigation (01.12.2018)
Published in The Journal of clinical investigation (01.12.2018)
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Journal Article
Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
Bertin, Berangere, Veron, Philippe, Leborgne, Christian, Deschamps, Jack-Yves, Moullec, Sophie, Fromes, Yves, Collaud, Fanny, Boutin, Sylvie, Latournerie, Virginie, van Wittenberghe, Laetitia, Delache, Benoit, Le Grand, Roger, Dereuddre-Bosquet, Nathalie, Benveniste, Olivier, Moullier, Philippe, Masurier, Carole, Merten, Otto, Mingozzi, Federico
Published in Scientific reports (21.01.2020)
Published in Scientific reports (21.01.2020)
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Journal Article
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
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
Published in Molecular therapy. Methods & clinical development (15.06.2018)
Published in Molecular therapy. Methods & clinical development (15.06.2018)
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Journal Article
Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients
Leborgne, Christian, Latournerie, Virginie, Boutin, Sylvie, Desgue, Diana, Quéré, Aliénor, Pignot, Elodie, Collaud, Fanny, Charles, Séverine, Simon Sola, Marcelo, Masat, Elisa, Jouen, Fabienne, Boyer, Olivier, Masurier, Carole, Mingozzi, Federico, Veron, Philippe
Published in Cellular immunology (01.08.2019)
Published in Cellular immunology (01.08.2019)
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Journal Article
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
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, 3rd, William J, Gross, David A, Boyer, Olivier, Anguela, Xavier M, Armour, Sean M, Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico
Published in Nature communications (04.11.2021)
Published in Nature communications (04.11.2021)
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Journal Article
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
Leborgne, Christian, Barbon, Elena, Alexander, Jeffrey M, Hanby, Hayley, Delignat, Sandrine, Cohen, Daniel M, Collaud, Fanny, Muraleetharan, Saghana, Lupo, Dan, Silverberg, Joseph, Huang, Karen, van Wittengerghe, Laetitia, Marolleau, Béatrice, Miranda, Adeline, Fabiano, Anna, Daventure, Victoria, Beck, Heena, Anguela, Xavier M, Ronzitti, Giuseppe, Armour, Sean M, Lacroix-Desmazes, Sebastien, Mingozzi, Federico
Published in Nature Medicine (01.07.2020)
Published in Nature Medicine (01.07.2020)
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AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
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
Published in Molecular therapy. Methods & clinical development (15.03.2019)
Published in Molecular therapy. Methods & clinical development (15.03.2019)
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Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors
Meliani, Amine, Boisgerault, Florence, Fitzpatrick, Zachary, Marmier, Solenne, Leborgne, Christian, Collaud, Fanny, Simon Sola, Marcelo, Charles, Severine, Ronzitti, Giuseppe, Vignaud, Alban, van Wittenberghe, Laetitia, Marolleau, Beatrice, Jouen, Fabienne, Tan, Sisareuth, Boyer, Olivier, Christophe, Olivier, Brisson, Alain R., Maguire, Casey A., Mingozzi, Federico
Published in Blood advances (24.10.2017)
Published in Blood advances (24.10.2017)
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Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors
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
Published in EMBO molecular medicine (07.04.2020)
Published in EMBO molecular medicine (07.04.2020)
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Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9
Lisjak, Michela, Iaconcig, Alessandra, Guarnaccia, Corrado, Vicidomini, Antonio, Moretti, Laura, Collaud, Fanny, Ronzitti, Giuseppe, Zentilin, Lorena, Muro, Andrés F.
Published in Molecular therapy. Methods & clinical development (14.12.2023)
Published in Molecular therapy. Methods & clinical development (14.12.2023)
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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
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
Published in Molecular therapy. Methods & clinical development (15.03.2019)
Published in Molecular therapy. Methods & clinical development (15.03.2019)
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Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle
Poupiot, Jérôme, Costa Verdera, Helena, Hardet, Romain, Colella, Pasqualina, Collaud, Fanny, Bartolo, Laurent, Davoust, Jean, Sanatine, Peggy, Mingozzi, Federico, Richard, Isabelle, Ronzitti, Giuseppe
Published in Molecular therapy. Methods & clinical development (13.12.2019)
Published in Molecular therapy. Methods & clinical development (13.12.2019)
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Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
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
Published in EBioMedicine (01.11.2020)
Published in EBioMedicine (01.11.2020)
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Journal Article
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
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
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
Shi, Xiaoxia, Aronson, Sem J., ten Bloemendaal, Lysbeth, Duijst, Suzanne, Bakker, Robert S., de Waart, Dirk R., Bortolussi, Giulia, Collaud, Fanny, Oude Elferink, Ronald P., Muro, Andrés F., Mingozzi, Federico, Ronzitti, Giuseppe, Bosma, Piter J.
Published in Molecular therapy. Methods & clinical development (12.03.2021)
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector
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.
Published in Molecular therapy. Methods & clinical development (12.03.2021)
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene
Aronson, Sem J., Bakker, Robert S., Moenis, Sascha, van Dijk, Remco, Bortolussi, Giulia, Collaud, Fanny, Shi, Xiaoxia, Duijst, Suzanne, ten Bloemendaal, Lysbeth, Ronzitti, Giuseppe, Muro, Andrés F., Mingozzi, Federico, Beuers, Ulrich, Bosma, Piter J.
Published in Molecular therapy. Methods & clinical development (11.09.2020)
Published in Molecular therapy. Methods & clinical development (11.09.2020)
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Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics
Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico
Published in Molecular therapy. Nucleic acids (29.11.2016)
Published in Molecular therapy. Nucleic acids (29.11.2016)
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Journal Article
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
Leborgne, Christian, Barbon, Elena, Alexander, Jeffrey, Hanby, Hayley, Delignat, Sandrine, Cohen, Daniel, Collaud, Fanny, Muraleetharan, Saghana, Lupo, Dan, Silverberg, Joseph, Huang, Karen, van Wittengerghe, Laetitia, Marolleau, Béatrice, Miranda, Adeline, Fabiano, Anna, Daventure, Victoria, Beck, Heena, Anguela, Xavier, Ronzitti, Giuseppe, Armour, Sean, Lacroix-Desmazes, Sebastien, Mingozzi, Federico
Published in Nature Medicine (01.06.2020)
Published in Nature Medicine (01.06.2020)
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