|
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
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
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
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
Journal articles
hal-04455812v1
|
|
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 articles
hal-04302850v1
|
|
Gene Therapy in Patients with the Crigler–Najjar Syndrome
Lorenzo D’antiga
,
Ulrich Beuers
,
Giuseppe Ronzitti
,
Nicola Brunetti-Pierri
,
Ulrich Baumann
Journal articles
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 Vidal
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
Journal articles
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 Sanmiguel
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
Journal articles
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 Bakker
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 articles
hal-03230901v1
|
|
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
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
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
Journal articles
hal-04324316v1
|
|
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
|
|
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
|
|
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
|
|
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
Journal articles
hal-04324265v1
|
|
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
|
|
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
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
Journal articles
hal-04324352v1
|
|
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
Giuseppe Ronzitti
,
David-Alexandre Gross
,
Federico Mingozzi
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
Journal articles
hal-04324362v1
|
|
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
Journal articles
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 Delignat
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
Journal articles
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 Collaud
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
Journal articles
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 Bordet
Journal articles
hal-02179298v1
|
|
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
|
|
Progress and challenges of gene therapy for Pompe disease
Giuseppe Ronzitti
,
Fanny Collaud
,
Pascal Laforet
,
Federico Mingozzi
Journal articles
hal-04324419v1
|
|
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
|
|
Progress and challenges of gene therapy for Pompe disease
G. Ronzitti
,
F. Collaud
,
P. Laforet
,
F. Mingozzi
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
Journal articles
hal-02880785v1
|
|
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
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
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
Journal articles
hal-04324412v1
|
|
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
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
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
Journal articles
hal-04324439v1
|
|
Combination Therapy Is the New Gene Therapy?
G. Ronzitti
,
F. Mingozzi
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
Journal articles
hal-04495633v1
|
|
Emerging Issues in AAV-Mediated In Vivo Gene Therapy
Pasqualina Colella
,
Giuseppe Ronzitti
,
Federico Mingozzi
Journal articles
hal-01812088v1
|
|
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Zachary Fitzpatrick
,
Christian Leborgne
,
Elena Barbon
,
Elisa Masat
,
Giuseppe Ronzitti
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
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 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
Journal articles
hal-02332988v1
|
|
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
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 articles
hal-04324436v1
|
|
Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors
M. Hosel
,
A. Huber
,
S. Bohlen
,
J. Lucifora
,
G. Ronzitti
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
Journal articles
hal-04324464v1
|
|
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
|
|
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
Journal articles
hal-02377508v1
|
|
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
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
Journal articles
hal-04324460v1
|
|
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
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
Journal articles
hal-04324476v1
|
|
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
|
|
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
Journal articles
hal-04324471v1
|
|
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
|