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Giuseppe Ronzitti

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Publications

Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9

Michela Lisjak , Alessandra Iaconcig , Corrado Guarnaccia , Antonio Vicidomini , Laura Moretti
Molecular Therapy - Methods and Clinical Development, 2023, 31, pp.101103. ⟨10.1016/j.omtm.2023.08.022⟩
Journal articles hal-04324300v1

Generation of three induced pluripotent stem cell lines from patients with glycogen storage disease type III

Lucille Rossiaud , Emilie Pellier , Manon Benabides , Xavier Nissan , Giuseppe Ronzitti
Stem Cell Research, 2023, 72, pp.103214. ⟨10.1016/j.scr.2023.103214⟩
Journal articles hal-04484199v1

Pathological modeling of glycogen storage disease type III with CRISPR/Cas9 edited human pluripotent stem cells

Lucille Rossiaud , Pascal Fragner , Elena Barbon , Antoine Gardin , Manon Benabides
Frontiers in Cell and Developmental Biology, 2023, 11, ⟨10.3389/fcell.2023.1163427⟩
Journal articles hal-04484270v1

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

Elena Barbon , Charlotte Kawecki , Solenne Marmier , Aboud Sakkal , Fanny Collaud
Gene Therapy, 2023, 30 (3-4), pp.245-254. ⟨10.1038/s41434-020-00218-6⟩
Journal articles hal-04455812v1
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Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

P. Sellier , P. Vidal , B. Bertin , E. Gicquel , E Bertil‐froidevaux
Journal of Inherited Metabolic Disease, 2023, ⟨10.1002/jimd.12625⟩
Journal articles hal-04302850v1

Gene Therapy in Patients with the Crigler–Najjar Syndrome

Lorenzo D’antiga , Ulrich Beuers , Giuseppe Ronzitti , Nicola Brunetti-Pierri , Ulrich Baumann
New England Journal of Medicine, 2023, 389 (7), pp.620-631. ⟨10.1056/NEJMoa2214084⟩
Journal articles hal-04324294v1
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A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III

Antoine Gardin , Jérémy Rouillon , Valle Montalvo-Romeral , Lucille Rossiaud , Patrice Vidal
The Journal of clinical investigation, 2023, 134 (2), pp.e172018. ⟨10.1172/JCI172018⟩
Journal articles hal-04387513v1

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

Jihad El Andari , Edith Renaud-Gabardos , Warut Tulalamba , Jonas Weinmann , Louise Mangin
Science Advances , 2022, 8 (38)
Journal articles hal-04273243v1

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
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Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques

Nerea Zabaleta , Urja Bhatt , Cécile Hérate , Pauline Maisonnasse , Julio Sanmiguel
Molecular Therapy, 2022, 30, pp.2952 - 2967. ⟨10.1016/j.ymthe.2022.05.007⟩
Journal articles hal-04510127v1

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
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Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients

Xiaoxia Shi , Sem Aronson , Lysbeth ten Bloemendaal , Suzanne Duijst , Robert Bakker
Molecular Therapy - Methods and Clinical Development, 2021, 20, pp.287-297. ⟨10.1016/j.omtm.2020.11.016⟩
Journal articles hal-04324276v1

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

Elena Barbon , Charlotte Kawecki , Solenne Marmier , Aboud Sakkal , Fanny Collaud
Gene Therapy, 2021, 30 (3-4), pp.245-254. ⟨10.1038/s41434-020-00218-6⟩
Journal articles hal-04324328v1

Thérapie génique, une nouvelle stratégie pour l’hypophosphatémie liée à l’X

V. Zhukouskaya , L. Jauze , S. Charles , C. Leborgne , S. Hilliquin
Annales d'Endocrinologie = Annals of Endocrinology, 2021, 82 (5), pp.255-256. ⟨10.1016/j.ando.2021.08.012⟩
Journal articles inserm-03413842v1

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

Giulia de Sabbata , Florence Boisgerault , Corrado Guarnaccia , Alessandra Iaconcig , Giulia Bortolussi
Molecular Therapy - Methods and Clinical Development, 2021, 20, pp.169-180. ⟨10.1016/j.omtm.2020.11.005⟩
Journal articles hal-04324316v1
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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
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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
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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

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

Helena Costa-Verdera , Fanny Collaud , Christopher Riling , Pauline Sellier , Jayme Nordin
Nature Communications, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩
Journal articles hal-04324265v1
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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
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SINEUP non-coding RNA targeting GDNF rescues motor deficits and neurodegeneration in a mouse model of Parkinson's disease

S. Espinoza , M. Scarpato , D. Damiani , F. Manago , M. Mereu
Molecular Therapy, 2020, 28, pp.642-652. ⟨10.1016/j.ymthe.2019.08.005⟩
Journal articles hal-02880797v1

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 (9), pp.2056-2072. ⟨10.1016/j.ymthe.2020.05.025⟩
Journal articles hal-04324352v1

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

Giuseppe Ronzitti , David-Alexandre Gross , Federico Mingozzi
Frontiers in Immunology, 2020, 11, ⟨10.3389/fimmu.2020.00670⟩
Journal articles hal-04363711v1

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

Christian Leborgne , Elena Barbon , Jeffrey Alexander , Hayley Hanby , Sandrine Delignat
Nature Medicine, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Journal articles hal-04324362v1
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A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

Sem Aronson , Robert Bakker , Sascha Moenis , Remco van Dijk , Giulia Bortolussi
Molecular Therapy - Methods and Clinical Development, 2020, 18, pp.250-258. ⟨10.1016/j.omtm.2020.06.002⟩
Journal articles hal-04324346v1
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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

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-04324427v1
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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

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-04324398v1
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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
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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

Progress and challenges of gene therapy for Pompe disease

Giuseppe Ronzitti , Fanny Collaud , Pascal Laforet , Federico Mingozzi
Annals of translational medicine, 2019, 7 (13), pp.287-287. ⟨10.21037/atm.2019.04.67⟩
Journal articles hal-04324419v1
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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

Progress and challenges of gene therapy for Pompe disease

G. Ronzitti , F. Collaud , P. Laforet , F. Mingozzi
Ann Transl Med, 2019, 7, pp.287. ⟨10.21037/atm.2019.04.67⟩
Journal articles hal-02880815v1

Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice

S. J. Aronson , R. S. Bakker , X. Shi , S. Duijst , L. ten Bloemendaal
J Hepatol, 2019, 71, pp.153-162. ⟨10.1016/j.jhep.2019.03.021⟩
Journal articles hal-02880785v1
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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

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

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 hal-04324412v1
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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-04324422v1

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-04324446v1

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

Amine Meliani , Florence Boisgerault , Romain Hardet , Solenne Marmier , Fanny Collaud
Nature Communications, 2018, 9 (1), pp.4098. ⟨10.1038/s41467-018-06621-3⟩
Journal articles hal-04324439v1

Combination Therapy Is the New Gene Therapy?

G. Ronzitti , F. Mingozzi
Molecular Therapy, 2018, 26, pp.12-14. ⟨10.1016/j.ymthe.2017.12.008⟩
Journal articles hal-02880814v1

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

Amine Meliani , Florence Boisgerault , Romain Hardet , Solenne Marmier , Fanny Collaud
Nature Communications, 2018, 9 (1), pp.4098. ⟨10.1038/s41467-018-06621-3⟩
Journal articles hal-04495633v1
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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
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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-04324453v1

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

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
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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

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-04324436v1
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Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors

M. Hosel , A. Huber , S. Bohlen , J. Lucifora , G. Ronzitti
Hepatology, 2017, 66 (1), pp.252-265. ⟨10.1002/hep.29176⟩
Journal articles hal-02880988v1

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

Francesco Puzzo , Pasqualina Colella , Maria Biferi , Deeksha Bali , Nicole Paulk
Science Translational Medicine, 2017, 9 (418), ⟨10.1126/scitranslmed.aam6375⟩
Journal articles hal-04324464v1
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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
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Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid alpha-glucosidase

F. Puzzo , P. Colella , M. G. Biferi , D. Bali , N. K. Paulk
Science Translational Medicine, 2017, 9 (418), pp.aam6375. ⟨10.1126/scitranslmed.aam6375⟩
Journal articles hal-02881011v1

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

Amine Meliani , Florence Boisgerault , Zachary Fitzpatrick , Solenne Marmier , Christian Leborgne
Blood Advances, 2017, 1 (23), pp.2019-2031. ⟨10.1182/bloodadvances.2017010181⟩
Journal articles hal-04324460v1
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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

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, pp.16049. ⟨10.1038/mtm.2016.49⟩
Journal articles hal-04324476v1
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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

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-04324471v1
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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