Hasil Pencarian - Severine Charles

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  1. 1
    Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

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

    Diterbitkan 2018-06-01
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  2. 2
    AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

    AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice oleh Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, Francesco Puzzo, Laetitia van Wittenberghe, Nicolas Guerchet, Nathalie Daniele, Bernard Gjata, Solenne Marmier, Severine Charles, Marcelo Simon Sola, Isabella Ragone, Christian Leborgne, Fanny Collaud, Federico Mingozzi

    Diterbitkan 2019-03-01
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  3. 3
    Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors

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

    Diterbitkan 2020-04-01
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  4. 4
    A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

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

    Diterbitkan 2016-01-01
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  5. 5
    Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector

    Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector oleh Giulia De Sabbata, Florence Boisgerault, Corrado Guarnaccia, Alessandra Iaconcig, Giulia Bortolussi, Fanny Collaud, Giuseppe Ronzitti, Marcelo Simon Sola, Patrice Vidal, Jeremy Rouillon, Severine Charles, Emanuele Nicastro, Lorenzo D’Antiga, Petr Ilyinskii, Federico Mingozzi, Takashi Kei Kishimoto, Andrés F. Muro

    Diterbitkan 2021-03-01
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  6. 6
    Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

    Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects oleh Pasqualina Colella, Pauline Sellier, Manuel J. Gomez, Maria G. Biferi, Guillaume Tanniou, Nicolas Guerchet, Mathilde Cohen-Tannoudji, Maryse Moya-Nilges, Laetitia van Wittenberghe, Natalie Daniele, Bernard Gjata, Jacomina Krijnse-Locker, Fanny Collaud, Marcelo Simon-Sola, Severine Charles, Umut Cagin, Federico Mingozzi

    Diterbitkan 2020-11-01
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  7. 7
    Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

    Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors oleh 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

    Diterbitkan 2017-10-01
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  8. 8
    Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

    Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration oleh Amine Meliani, Florence Boisgerault, Romain Hardet, Solenne Marmier, Fanny Collaud, Giuseppe Ronzitti, Christian Leborgne, Helena Costa Verdera, Marcelo Simon Sola, Severine Charles, Alban Vignaud, Laetitia van Wittenberghe, Giorgia Manni, Olivier Christophe, Francesca Fallarino, Christopher Roy, Alicia Michaud, Petr Ilyinskii, Takashi Kei Kishimoto, Federico Mingozzi

    Diterbitkan 2018-10-01
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  9. 9
    Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

    Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome oleh Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’h, Sem J. Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G. Dufour, Florence Lacoste, Cyril Luc, Laetitia v. Wittenberghe, Samia Martin, Christine Le Bec, Piter J. Bosma, Andres F. Muro, Giuseppe Ronzitti, Matthias Hebben, Federico Mingozzi

    Diterbitkan 2019-03-01
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  10. 10
    Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates

    Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates oleh Helena Costa-Verdera, Fanny Collaud, Christopher R. Riling, Pauline Sellier, Jayme M. L. Nordin, G. Michael Preston, Umut Cagin, Julien Fabregue, Simon Barral, Maryse Moya-Nilges, Jacomina Krijnse-Locker, Laetitia van Wittenberghe, Natalie Daniele, Bernard Gjata, Jeremie Cosette, Catalina Abad, Marcelo Simon-Sola, Severine Charles, Mathew Li, Marco Crosariol, Tom Antrilli, William J. Quinn, David A. Gross, Olivier Boyer, Xavier M. Anguela, Sean M. Armour, Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi

    Diterbitkan 2021-11-01
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