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Ronzitti Giuseppe - INTEGRARE

Journal articles23 documents

  • David-Alexandre Gross, Novella Tedesco, Christian Leborgne, Giuseppe Ronzitti. Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients. Frontiers in Immunology, Frontiers, 2022, 13, ⟨10.3389/fimmu.2022.857276⟩. ⟨hal-03640310⟩
  • Helena Costa-Verdera, Fanny Collaud, Christopher Riling, Pauline Sellier, Jayme Nordin, et al.. Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates. Nature Communications, Nature Publishing Group, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩. ⟨hal-03449262⟩
  • Volha Zhukouskaya, Louisa Jauze, Séverine Charles, Christian Leborgne, Stéphane Hilliquin, et al.. AAV liver gene therapy-mediated inhibition of FGF23 signaling as a therapeutic strategy for X-linked hypophosphatemia. Endocrine Abstracts, Elsevier Science, 2021, ⟨10.1530/endoabs.73.OC6.2⟩. ⟨inserm-03413999⟩
  • Édouard Berling, Pascal Laforêt, Karim Wahbi, Philippe H. Labrune, François Michael Petit, et al.. Narrative review of glycogen storage disorder type III with a focus on neuromuscular, cardiac and therapeutic aspects. Journal of Inherited Metabolic Disease, Springer Verlag, 2021, 44 (3), pp.521-533. ⟨10.1002/jimd.12355⟩. ⟨hal-03230901⟩
  • Marcelo Simon Sola, Takashi Kei Kishimoto, Giulia de Sabbata, Florence Boisgérault, Corrado Guarnaccia, et al.. Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2021, 20, pp.169 - 180. ⟨10.1016/j.omtm.2020.11.005⟩. ⟨hal-03193757⟩
  • Christian Leborgne, Elena Barbon, Jeffrey Alexander, Hayley Hanby, Sandrine Delignat, et al.. IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies. Nature Medicine, Nature Publishing Group, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩. ⟨hal-03031762⟩
  • Umut Cagin, Francesco Puzzo, Manuel Jose Gomez, Maryse Moya-Nilges, Pauline Sellier, et al.. Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase. Molecular Therapy, Cell Press, 2020, 28, pp.2056 - 2072. ⟨10.1016/j.ymthe.2020.05.025⟩. ⟨hal-03491957⟩
  • Laurent Bartolo, Stéphanie Li Chung Tong, Pascal Chappert, Dominique Urbain, Fanny Collaud, et al.. Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity. JCI Insight, American Society for Clinical Investigation, 2019, 4 (11), pii: 127008. ⟨10.1172/jci.insight.127008⟩. ⟨hal-02332827⟩
  • Louisa Jauze, Laure Monteillet, Gilles Mithieux, Fabienne Rajas, Giuseppe Ronzitti. Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III. Human Gene Therapy, Mary Ann Liebert, 2019, 30 (10), pp.1263-1273. ⟨10.1089/hum.2019.102⟩. ⟨inserm-02379154⟩
  • Pascal Laforêt, Michio Inoue, Evelyne Goillot, Claire Lefeuvre, Umut Cagin, et al.. Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment. Acta Neuropathologica Communications, BioMed Central part of Springer Science, 2019, 7 (1), ⟨10.1186/s40478-019-0815-2⟩. ⟨hal-02414161⟩
  • Jérôme Poupiot, Helena Costa Verdera, Romain Hardet, Pasqualina Colella, Fanny Collaud, et al.. Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2019, 15, pp.83 - 100. ⟨10.1016/j.omtm.2019.08.012⟩. ⟨hal-02392771⟩
  • Nicola Salvatore Orefice, Benoît Souchet, Jérôme Braudeau, Sandro Alves, Françoise Piguet, et al.. Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2019, 14, pp.237 - 251. ⟨10.1016/j.omtm.2019.06.005⟩. ⟨cea-02997523⟩
  • Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc'H, Sem Aronson, Thierry Bordet, et al.. Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2019, 12, pp.157-174. ⟨10.1016/j.omtm.2018.12.011⟩. ⟨hal-02179298⟩
  • Florence Boisgérault, Amine Meliani, Romain Hardet, Solenne Marmier, Fanny Collaud, et al.. Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration. Nature Communications, Nature Publishing Group, 2018, 9 (1), pp.4098. ⟨10.1038/s41467-018-06621-3⟩. ⟨hal-02177709⟩
  • Klaudia Kuranda, Priscilla Jean-Alphonse, Christian Leborgne, Romain Hardet, Fanny Collaud, et al.. Exposure to wild-type AAV drives distinct capsid immunity profiles in humans. Journal of Clinical Investigation, American Society for Clinical Investigation, 2018, 128 (12), pp.5267-5279. ⟨10.1172/JCI122372⟩. ⟨hal-01977027⟩
  • Patrice Vidal, Serena Pagliarani, Pasqualina Colella, Helena Costa Verdera, Louisa Jauze, et al.. Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression. Molecular Therapy, Cell Press, 2018, 26 (3), pp.890-901. ⟨10.1016/j.ymthe.2017.12.019⟩. ⟨hal-02332988⟩
  • Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi. Emerging Issues in AAV-Mediated In Vivo Gene Therapy. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2018, 8, pp.87-104. ⟨10.1016/j.omtm.2017.11.007⟩. ⟨hal-01812088⟩
  • Zachary Fitzpatrick, Christian Leborgne, Elena Barbon, Elisa Masat, Giuseppe Ronzitti, et al.. Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2018, 9, pp.119-129. ⟨10.1016/j.omtm.2018.02.003⟩. ⟨hal-01830017⟩
  • Amine Meliani, Florence Boisgérault, Zachary Fitzpatrick, Solenne Marmier, Christian Leborgne, et al.. Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors. Blood Advances, The American Society of Hematology, 2017, 1 (23), pp.2019-2031. ⟨10.1182/bloodadvances.2017010181⟩. ⟨hal-02377508⟩
  • Sang-Oh Han, Giuseppe Ronzitti, Benjamin Arnson, Christian Leborgne, Songtao Li, et al.. Low-dose liver targeted gene therapy for Pompe disease enhances therapeutic efficacy of ERT via immune tolerance induction. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2017, ⟨10.1016/j.omtm.2016.12.010⟩. ⟨hal-01432085⟩
  • Elena Barbon, Mattia Ferrarese, Laetitia van Wittenberghe, Peggy Sanatine, Giuseppe Ronzitti, et al.. Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics . Molecular Therapy - Nucleic Acids, Elsevier, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩. ⟨hal-01449506⟩
  • Elisa Masat, Pascal Laforêt, Marie de Antonio, Guillaume Corre, Barbara Perniconi, et al.. Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients. Scientific Reports, Nature Publishing Group, 2016, 6, pp.36182. ⟨10.1038/srep36182⟩. ⟨hal-01404918⟩
  • Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, Fanny Collaud, Severine Charles, et al.. A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. Molecular Therapy - Methods and Clinical Development, Nature Publishing Group, 2016, 3, ⟨10.1038/mtm.2016.49⟩. ⟨hal-01474146⟩