Access content directly

Giuseppe Ronzitti

Researcher identifiers



Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

David-Alexandre Gross , Novella Tedesco , Christian Leborgne , Giuseppe Ronzitti
Frontiers in Immunology, 2022, 13, ⟨10.3389/fimmu.2022.857276⟩
Journal articles hal-03640310v1

Narrative review of glycogen storage disorder type III with a focus on neuromuscular, cardiac and therapeutic aspects

Édouard Berling , Pascal Laforêt , Karim Wahbi , Philippe H. Labrune , François Michael Petit
Journal of Inherited Metabolic Disease, 2021, 44 (3), pp.521-533. ⟨10.1002/jimd.12355⟩
Journal articles hal-03230901v1

AAV liver gene therapy-mediated inhibition of FGF23 signaling as a therapeutic strategy for X-linked hypophosphatemia

Volha Zhukouskaya , Louisa Jauze , Séverine Charles , Christian Leborgne , Stéphane Hilliquin
Endocrine Abstracts, 2021, ⟨10.1530/endoabs.73.OC6.2⟩
Journal articles inserm-03413999v1
Image document

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

Marcelo Simon Sola , Takashi Kei Kishimoto , Giulia de Sabbata , Florence Boisgérault , Corrado Guarnaccia
Molecular Therapy - Methods and Clinical Development, 2021, 20, pp.169 - 180. ⟨10.1016/j.omtm.2020.11.005⟩
Journal articles hal-03193757v1
Image document

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

Helena Costa-Verdera , Fanny Collaud , Christopher R Riling , Pauline Sellier , Jayme M L Nordin
Nature Communications, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩
Journal articles hal-03449262v1
Image document

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

Volha Zhukouskaya , Louisa Jauze , Séverine Charles , Christian Leborgne , Stéphane Hilliquin
Science Advances , 2021, 7 (44), pp.eabj5018. ⟨10.1126/sciadv.abj5018⟩
Journal articles inserm-03687057v1
Image document

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

Umut Cagin , Francesco Puzzo , Manuel Jose Gomez , Maryse Moya-Nilges , Pauline Sellier
Molecular Therapy, 2020, 28, pp.2056 - 2072. ⟨10.1016/j.ymthe.2020.05.025⟩
Journal articles hal-03491957v1
Image document

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Christian Leborgne , Elena Barbon , Jeffrey M Alexander , Hayley Hanby , Sandrine Delignat
Nature Medicine, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Journal articles hal-03031762v1
Image document

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

Fanny Collaud , Giulia Bortolussi , Laurence Guianvarc'H , Sem Aronson , Thierry Bordet
Molecular Therapy - Methods and Clinical Development, 2019, 12, pp.157-174. ⟨10.1016/j.omtm.2018.12.011⟩
Journal articles hal-02179298v1
Image document

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle

Jérôme Poupiot , Helena Costa Verdera , Romain Hardet , Pasqualina Colella , Fanny Collaud
Molecular Therapy - Methods and Clinical Development, 2019, 15, pp.83 - 100. ⟨10.1016/j.omtm.2019.08.012⟩
Journal articles hal-02392771v1

Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment

Pascal Laforêt , Michio Inoue , Evelyne Goillot , Claire Lefeuvre , Umut Cagin
Acta Neuropathologica Communications, 2019, 7 (1), ⟨10.1186/s40478-019-0815-2⟩
Journal articles hal-02414161v1
Image document

Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III

Louisa Jauze , Laure Monteillet , Gilles Mithieux , Fabienne Rajas , Giuseppe Ronzitti
Human Gene Therapy, 2019, 30 (10), pp.1263-1273. ⟨10.1089/hum.2019.102⟩
Journal articles inserm-02379154v1

Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity

Laurent Bartolo , Stéphanie Li Chung Tong , Pascal Chappert , Dominique Urbain , Fanny Collaud
JCI Insight, 2019, 4 (11), pii: 127008. ⟨10.1172/jci.insight.127008⟩
Journal articles hal-02332827v1
Image document

Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain

Nicola Salvatore Orefice , Benoît Souchet , Jérôme Braudeau , Sandro Alves , Françoise Piguet
Molecular Therapy - Methods and Clinical Development, 2019, 14, pp.237 - 251. ⟨10.1016/j.omtm.2019.06.005⟩
Journal articles cea-02997523v1

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

Florence Boisgérault , Amine Meliani , Romain Hardet , Solenne Marmier , Fanny Collaud
Nature Communications, 2018, 9 (1), pp.4098. ⟨10.1038/s41467-018-06621-3⟩
Journal articles hal-02177709v1
Image document

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

Pasqualina Colella , Giuseppe Ronzitti , Federico Mingozzi
Molecular Therapy - Methods and Clinical Development, 2018, 8, pp.87-104. ⟨10.1016/j.omtm.2017.11.007⟩
Journal articles hal-01812088v1
Image document

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

Zachary Fitzpatrick , Christian Leborgne , Elena Barbon , Elisa Masat , Giuseppe Ronzitti
Molecular Therapy - Methods and Clinical Development, 2018, 9, pp.119-129. ⟨10.1016/j.omtm.2018.02.003⟩
Journal articles hal-01830017v1

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression

Patrice Vidal , Serena Pagliarani , Pasqualina Colella , Helena Costa Verdera , Louisa Jauze
Molecular Therapy, 2018, 26 (3), pp.890-901. ⟨10.1016/j.ymthe.2017.12.019⟩
Journal articles hal-02332988v1

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

Klaudia Kuranda , Priscilla Jean-Alphonse , Christian Leborgne , Romain Hardet , Fanny Collaud
Journal of Clinical Investigation, 2018, 128 (12), pp.5267-5279. ⟨10.1172/JCI122372⟩
Journal articles hal-01977027v1
Image document

Low-dose liver targeted gene therapy for Pompe disease enhances therapeutic efficacy of ERT via immune tolerance induction

Sang-Oh Han , Giuseppe Ronzitti , Benjamin Arnson , Christian Leborgne , Songtao Li
Molecular Therapy - Methods and Clinical Development, 2017, ⟨10.1016/j.omtm.2016.12.010⟩
Journal articles hal-01432085v1

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

Amine Meliani , Florence Boisgérault , Zachary Fitzpatrick , Solenne Marmier , Christian Leborgne
Blood Advances, 2017, 1 (23), pp.2019-2031. ⟨10.1182/bloodadvances.2017010181⟩
Journal articles hal-02377508v1
Image document

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

Elena Barbon , Mattia Ferrarese , Laetitia van Wittenberghe , Peggy Sanatine , Giuseppe Ronzitti
Molecular Therapy - Nucleic Acids, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩
Journal articles hal-01449506v1
Image document

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Elisa Masat , Pascal Laforêt , Marie de Antonio , Guillaume Corre , Barbara Perniconi
Scientific Reports, 2016, 6, pp.36182. ⟨10.1038/srep36182⟩
Journal articles hal-01404918v1
Image document

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

Giuseppe Ronzitti , Giulia Bortolussi , Remco van Dijk , Fanny Collaud , Severine Charles
Molecular Therapy - Methods and Clinical Development, 2016, 3, ⟨10.1038/mtm.2016.49⟩
Journal articles hal-01474146v1