Giuseppe Ronzitti

Identifiants chercheurs

IdHAL :

giuseppe-ronzitti

ORCID :

0000-0002-9184-0210

IdRef :

26470309X

VIAF :

133166597331517592666

ResearcherID :

http://www.researcherid.com/rid/C-2566-2018

Présentation

Publications

Article dans une revue (62)

Enhanced Lysosomal Glycogen Breakdown is associated with Liver Tumorigenesis in Glycogen Storage Disease Type III Valle Montalvo-Romeral,Louisa Jauze,Gwendoline Perrot,Mouna Amaouche,Antoine Gardinet al. JHEP Reports Innovation in Hepatology, 2026, 8 (3), pp.101702. ⟨10.1016/j.jhepr.2025.101702⟩ Article dans une revue hal-05428665v2
Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer Marine Laurent,Rim Harb,Christine Jenny,Julie Oustelandt,Simon Jimenezet al. Communications Medicine, 2025, 5 (1), pp.110. ⟨10.1038/s43856-025-00816-8⟩ Article dans une revue hal-05447606v1
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems Francesca Di Leva,Michele Arnoldi,Stefania Santarelli,Mathieu Massonot,Marianne Victoria Leméeet al. Molecular Therapy, 2025, 33 (3), pp.1180 - 1196. ⟨10.1016/j.ymthe.2024.12.043⟩ Article dans une revue hal-05038000v1
Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver Louisa Jauze,Mallaury Vie,Quentin Miagoux,Lucille Rossiaud,Patrice Vidalet al. JCI Insight, 2024, 9 (11), pp.e172614. ⟨10.1172/jci.insight.172614⟩ Article dans une revue hal-05216074v1
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I Xiaoxia Shi,Giulia Bortolussi,Fanny Collaud,Pierre-Romain Le Brun,Lysbeth Ten Bloemendaalet al. Molecular Therapy - Methods and Clinical Development, 2024, 32 (4), pp.101363. ⟨10.1016/j.omtm.2024.101363⟩ Article dans une revue hal-05216071v1
Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness Tiziana La Bella,Bérangère Bertin,Ante Mihaljevic,Justine Nozi,Patrice Vidalet al. Molecular Therapy - Methods and Clinical Development, 2024, 32 (3), pp.101327. ⟨10.1016/j.omtm.2024.101327⟩ Article dans une revue hal-04797131v1
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 Benabideset al. Frontiers in Cell and Developmental Biology, 2023, 11, ⟨10.3389/fcell.2023.1163427⟩ Article dans une revue hal-04484270v1
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 Morettiet al. Molecular Therapy - Methods and Clinical Development, 2023, 31, pp.101103. ⟨10.1016/j.omtm.2023.08.022⟩ Article dans une revue 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 Ronzittiet al. Stem Cell Research, 2023, 72, pp.103214. ⟨10.1016/j.scr.2023.103214⟩ Article dans une revue hal-04484199v1
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‐froidevauxet al. Journal of Inherited Metabolic Disease, 2023, 47 (1), pp.119-134. ⟨10.1002/jimd.12625⟩ Article dans une revue hal-04795500v1
Gene Therapy in Patients with the Crigler–Najjar Syndrome Lorenzo D’antiga,Ulrich Beuers,Giuseppe Ronzitti,Nicola Brunetti-Pierri,Ulrich Baumannet al. New England Journal of Medicine, 2023, 389 (7), pp.620-631. ⟨10.1056/NEJMoa2214084⟩ Article dans une revue hal-04324294v1
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 Vidalet al. The Journal of clinical investigation, 2023, 134 (2), pp.e172018. ⟨10.1172/JCI172018⟩ Article dans une revue hal-04387513v1
Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor Elena Barbon,Charlotte Kawecki,Solenne Marmier,Aboud Sakkal,Fanny Collaudet al. Gene Therapy, 2023, 30 (3-4), pp.245-254. ⟨10.1038/s41434-020-00218-6⟩ Article dans une revue hal-04455812v1
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⟩ Article dans une revue hal-03640310v1
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 Sanmiguelet al. Molecular Therapy, 2022, 30, pp.2952 - 2967. ⟨10.1016/j.ymthe.2022.05.007⟩ Article dans une revue hal-04510127v1
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 Manginet al. Science Advances , 2022, 8 (38), ⟨10.1126/sciadv.abn4704⟩ Article dans une revue hal-04273243v1
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 Guarnacciaet al. Molecular Therapy - Methods and Clinical Development, 2021, 20, pp.169 - 180. ⟨10.1016/j.omtm.2020.11.005⟩ Article dans une revue hal-03193757v1
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 Bortolussiet al. Molecular Therapy - Methods and Clinical Development, 2021, 20, pp.169-180. ⟨10.1016/j.omtm.2020.11.005⟩ Article dans une revue hal-04324316v1
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 Hilliquinet al. Endocrine Abstracts, 2021, ⟨10.1530/endoabs.73.OC6.2⟩ Article dans une revue inserm-03413999v1
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 Bakkeret al. Molecular Therapy - Methods and Clinical Development, 2021, 20, pp.287-297. ⟨10.1016/j.omtm.2020.11.016⟩ Article dans une revue hal-04324276v1
Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor Elena Barbon,Charlotte Kawecki,Solenne Marmier,Aboud Sakkal,Fanny Collaudet al. Gene Therapy, 2021, 30 (3-4), pp.245-254. ⟨10.1038/s41434-020-00218-6⟩ Article dans une revue (data paper) hal-04324328v1
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 Petitet al. Journal of Inherited Metabolic Disease, 2021, 44 (3), pp.521-533. ⟨10.1002/jimd.12355⟩ Article dans une revue hal-03230901v1
Thérapie génique, une nouvelle stratégie pour l’hypophosphatémie liée à l’X V. Zhukouskaya,L. Jauze,S. Charles,C. Leborgne,S. Hilliquinet al. Annales d'Endocrinologie = Annals of Endocrinology, 2021, 82 (5), pp.255-256. ⟨10.1016/j.ando.2021.08.012⟩ Article dans une revue inserm-03413842v1
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 Nordinet al. Nature Communications, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩ Article dans une revue hal-03449262v1
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 Hilliquinet al. Science Advances , 2021, 7 (44), pp.eabj5018. ⟨10.1126/sciadv.abj5018⟩ Article dans une revue inserm-03687057v1
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. Mereuet al. Molecular Therapy, 2020, 28, pp.642-652. ⟨10.1016/j.ymthe.2019.08.005⟩ Article dans une revue 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 Sellieret al. Molecular Therapy, 2020, 28, pp.2056 - 2072. ⟨10.1016/j.ymthe.2020.05.025⟩ Article dans une revue hal-03491957v1
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 Bortolussiet al. Molecular Therapy - Methods and Clinical Development, 2020, 18, pp.250-258. ⟨10.1016/j.omtm.2020.06.002⟩ Article dans une revue hal-04324346v1
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 Delignatet al. Nature Medicine, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩ Article dans une revue hal-03031762v1
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⟩ Article dans une revue hal-04363711v1
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⟩ Article dans une revue 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 Bloemendaalet al. J Hepatol, 2019, 71, pp.153-162. ⟨10.1016/j.jhep.2019.03.021⟩ Article dans une revue hal-02880785v1
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 Caginet al. Acta Neuropathologica Communications, 2019, 7 (1), ⟨10.1186/s40478-019-0815-2⟩ Article dans une revue hal-02414161v1
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⟩ Article dans une revue inserm-02379154v1
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⟩ Article dans une revue hal-04324419v1
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome Fanny Collaud,Giulia Bortolussi,Laurence Guianvarc’h,Sem Aronson,Thierry Bordetet al. Molecular Therapy - Methods and Clinical Development, 2019, 12, pp.157-174. ⟨10.1016/j.omtm.2018.12.011⟩ Article dans une revue hal-04324427v1
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 Collaudet al. Molecular Therapy - Methods and Clinical Development, 2019, 15, pp.83 - 100. ⟨10.1016/j.omtm.2019.08.012⟩ Article dans une revue 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 Collaudet al. Molecular Therapy - Methods and Clinical Development, 2019, 15, pp.83-100. ⟨10.1016/j.omtm.2019.08.012⟩ Article dans une revue hal-04324398v1
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome Fanny Collaud,Giulia Bortolussi,Laurence Guianvarc'H,Sem Aronson,Thierry Bordetet al. Molecular Therapy - Methods and Clinical Development, 2019, 12, pp.157-174. ⟨10.1016/j.omtm.2018.12.011⟩ Article dans une revue hal-02179298v1
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 Collaudet al. JCI Insight, 2019, 4 (11), pii: 127008. ⟨10.1172/jci.insight.127008⟩ Article dans une revue hal-04324422v1
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 Piguetet al. Molecular Therapy - Methods and Clinical Development, 2019, 14, pp.237-251. ⟨10.1016/j.omtm.2019.06.005⟩ Article dans une revue hal-04324412v1
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 Piguetet al. Molecular Therapy - Methods and Clinical Development, 2019, 14, pp.237 - 251. ⟨10.1016/j.omtm.2019.06.005⟩ Article dans une revue cea-02997523v1
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⟩ Article dans une revue hal-01812088v1
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration Amine Meliani,Florence Boisgerault,Romain Hardet,Solenne Marmier,Fanny Collaudet al. Nature Communications, 2018, 9 (1), pp.4098. ⟨10.1038/s41467-018-06621-3⟩ Article dans une revue hal-04495633v1
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction Zachary Fitzpatrick,Christian Leborgne,Elena Barbon,Elisa Masat,Giuseppe Ronzittiet al. Molecular Therapy - Methods and Clinical Development, 2018, 9, pp.119-129. ⟨10.1016/j.omtm.2018.02.003⟩ Article dans une revue 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 Collaudet al. Nature Communications, 2018, 9 (1), pp.4098. ⟨10.1038/s41467-018-06621-3⟩ Article dans une revue hal-04324439v1
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration Florence Boisgérault,Amine Meliani,Romain Hardet,Solenne Marmier,Fanny Collaudet al. Nature Communications, 2018, 9 (1), pp.4098. ⟨10.1038/s41467-018-06621-3⟩ Article dans une revue hal-02177709v1
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⟩ Article dans une revue hal-02880814v1
Exposure to wild-type AAV drives distinct capsid immunity profiles in humans Klaudia Kuranda,Priscilla Jean-Alphonse,Christian Leborgne,Romain Hardet,Fanny Collaudet al. Journal of Clinical Investigation, 2018, 128 (12), pp.5267-5279. ⟨10.1172/JCI122372⟩ Article dans une revue 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 Jauzeet al. Molecular Therapy, 2018, 26 (3), pp.890-901. ⟨10.1016/j.ymthe.2017.12.019⟩ Article dans une revue hal-02332988v1
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction Zachary Fitzpatrick,Christian Leborgne,Elena Barbon,Elisa Masat,Giuseppe Ronzittiet al. Molecular Therapy - Methods and Clinical Development, 2018, 9, pp.119-129. ⟨10.1016/j.omtm.2018.02.003⟩ Article dans une revue 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 Jauzeet al. Molecular Therapy, 2018, 26 (3), pp.890-901. ⟨10.1016/j.ymthe.2017.12.019⟩ Article dans une revue hal-04324453v1
Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors M. Hosel,A. Huber,S. Bohlen,J. Lucifora,G. Ronzittiet al. Hepatology, 2017, 66 (1), pp.252-265. ⟨10.1002/hep.29176⟩ Article dans une revue hal-02880988v1
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 Liet al. Molecular Therapy - Methods and Clinical Development, 2017, ⟨10.1016/j.omtm.2016.12.010⟩ Article dans une revue 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 Leborgneet al. Blood Advances, 2017, 1 (23), pp.2019-2031. ⟨10.1182/bloodadvances.2017010181⟩ Article dans une revue hal-02377508v1
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors Amine Meliani,Florence Boisgerault,Zachary Fitzpatrick,Solenne Marmier,Christian Leborgneet al. Blood Advances, 2017, 1 (23), pp.2019-2031. ⟨10.1182/bloodadvances.2017010181⟩ Article dans une revue hal-04324460v1
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. Paulket al. Science Translational Medicine, 2017, 9 (418), pp.aam6375. ⟨10.1126/scitranslmed.aam6375⟩ Article dans une revue hal-02881011v1
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 Ronzittiet al. Molecular Therapy - Nucleic Acids, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩ Article dans une revue hal-04324471v1
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 Perniconiet al. Scientific Reports, 2016, 6, pp.36182. ⟨10.1038/srep36182⟩ Article dans une revue 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 Ronzittiet al. Molecular Therapy - Nucleic Acids, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩ Article dans une revue 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 Charleset al. Molecular Therapy - Methods and Clinical Development, 2016, 3, pp.16049. ⟨10.1038/mtm.2016.49⟩ Article dans une revue hal-04324476v1
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 Charleset al. Molecular Therapy - Methods and Clinical Development, 2016, 3, ⟨10.1038/mtm.2016.49⟩ Article dans une revue hal-01474146v1