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Séverine Charles

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
2018 · Amine Meliani, Florence Boisgerault, et al. · Nature Communications · 272
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Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
2018 · Zachary Fitzpatrick, Christian Leborgne, et al. · Molecular Therapy — Methods & Clinical Development · 195
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Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
2017 · Francesco Puzzo, Pasqualina Colella, et al. · Science Translational Medicine · 141
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors
2017 · Amine Meliani, Florence Boisgerault, et al. · Blood Advances · 133
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
2018 · Pasqualina Colella, Pauline Sellier, et al. · Molecular Therapy — Methods & Clinical Development · 78
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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
2016 · Giuseppe Ronzitti, Giulia Bortolussi, et al. · Molecular Therapy — Methods & Clinical Development · 72
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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
2018 · Fanny Collaud, Giulia Bortolussi, et al. · Molecular Therapy — Methods & Clinical Development · 65
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Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients
2018 · Christian Leborgne, Virginie Latournerie, et al. · Cellular Immunology · 53
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Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor
2021 · Elena Barbon, Charlotte Kawecki, et al. · Gene Therapy · 44
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Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
2021 · Helena Costa-Verdera, Fanny Collaud, et al. · Nature Communications · 43
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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
2017 · Patrice Vidal, Serena Pagliarani, et al. · Molecular Therapy · 41
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Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome
2021 · Martina Parrini, Shovan Naskar, et al. · Molecular Therapy · 34
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Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors
2020 · Elena Barbon, Gabriel Ayme, et al. · EMBO Molecular Medicine · 28
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
2020 · Pasqualina Colella, Pauline Sellier, et al. · EBioMedicine · 26
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Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector
2020 · Giulia De Sabbata, Florence Boisgerault, et al. · Molecular Therapy — Methods & Clinical Development · 25
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Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase
2020 · Umut Cagin, Francesco Puzzo, et al. · Molecular Therapy · 25
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A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia
2021 · Volha V. Zhukouskaya, Louisa Jauze, et al. · Science Advances · 15
Real-time monitoring of cell transplantation in mouse dystrophic muscles by a secreted alkaline phosphatase reporter gene
2009 · X Gerard, L Vignaud, et al. · Gene Therapy · 10
A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia
2022 · Volha V Zhukouskaya, Louisa Jauze, et al. · Yearbook of pediatric endocrinology · 1
Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes
2017 · P. Colella, F. Puzzo, et al. · Neuromuscular Disorders · 1
Adeno associated vector-based gene therapy strategy for type 3 glycogen storage disease
2017 · P. Vidal, G. Ronzitti, et al. · Neuromuscular Disorders · 1
158. Cryptic ATG Removal from Synthetic Introns Increase the Therapeutic Efficacy of AAV Vector Mediated Gene Transfer
2016 · Giuseppe Ronzitti, Fanny Collaud, et al. · Molecular Therapy · 1
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Thérapie génique, une nouvelle stratégie pour l’hypophosphatémie liée à l’X
2021 · V. Zhukouskaya, L. Jauze, et al. · Annales d Endocrinologie
AAV liver gene therapy-mediated inhibition of FGF23 signaling as a therapeutic strategy for X-linked hypophosphatemia
2021 · Volha Zhukouskaya, Louisa Jauze, et al. · Endocrine Abstracts
Tandem promoter design confers tolerogenic and persistent transgene expression to AAV gene therapy in neonate Pompe mice
2019 · Pasqualina Colella, Pauline Sellier, et al. · Molecular Genetics and Metabolism
Works count: 30
Citation count: 1,304
H-index: 16
I10-index: 18
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