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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
Journal articles
hal-03640310v1
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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 articles
hal-03230901v1
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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
Journal articles
inserm-03413999v1
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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
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
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
Journal articles
inserm-03687057v1
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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
Journal articles
hal-03491957v1
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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
Journal articles
hal-03031762v1
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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
Journal articles
hal-02179298v1
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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
Journal articles
hal-02392771v1
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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
Journal articles
hal-02414161v1
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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
Journal articles
inserm-02379154v1
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Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity
Laurent Bartolo
,
Stéphanie Li Chung Tong
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Pascal Chappert
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Dominique Urbain
,
Fanny Collaud
Journal articles
hal-02332827v1
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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
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Benoît Souchet
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Jérôme Braudeau
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Sandro Alves
,
Françoise Piguet
Journal articles
cea-02997523v1
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Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
Florence Boisgérault
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Amine Meliani
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Romain Hardet
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Solenne Marmier
,
Fanny Collaud
Journal articles
hal-02177709v1
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Emerging Issues in AAV-Mediated In Vivo Gene Therapy
Pasqualina Colella
,
Giuseppe Ronzitti
,
Federico Mingozzi
Journal articles
hal-01812088v1
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Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Zachary Fitzpatrick
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Christian Leborgne
,
Elena Barbon
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Elisa Masat
,
Giuseppe Ronzitti
Journal articles
hal-01830017v1
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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
Patrice Vidal
,
Serena Pagliarani
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Pasqualina Colella
,
Helena Costa Verdera
,
Louisa Jauze
Journal articles
hal-02332988v1
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Exposure to wild-type AAV drives distinct capsid immunity profiles in humans
Klaudia Kuranda
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Priscilla Jean-Alphonse
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Christian Leborgne
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Romain Hardet
,
Fanny Collaud
Journal articles
hal-01977027v1
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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
Journal articles
hal-01432085v1
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Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors
Amine Meliani
,
Florence Boisgérault
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Zachary Fitzpatrick
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Solenne Marmier
,
Christian Leborgne
Journal articles
hal-02377508v1
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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
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Mattia Ferrarese
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Laetitia van Wittenberghe
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Peggy Sanatine
,
Giuseppe Ronzitti
Journal articles
hal-01449506v1
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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
Journal articles
hal-01404918v1
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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
Journal articles
hal-01474146v1
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