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

Christian Leborgne, Elena Barbon, J M Alexander, +19

Nature Medicine

Vivo Gene TherapyAutoimmune DiseaseIgg-cleaving Endopeptidase EnablesImmunologyAutoimmunity +6

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

Amine Meliani, Florence Boisgérault, Romain Hardet, +17

Nature Communications

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

Zachary Fitzpatrick, Christian Leborgne, Elena Barbon, +10

Molecular Therapy — Methods & Clinical Development

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

Francesco Puzzo, Pasqualina Colella, Maria Grazia Biferi, +22

Science Translational Medicine

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

Amine Meliani, Florence Boisgérault, Zachary Fitzpatrick, +16

Blood Advances

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

Klaudia Kuranda, Priscilla Jean-Alphonse, Christian Leborgne, +7

Journal of Clinical Investigation

Healthy SubjectsAdaptive Immune SystemHumoral ResponseImmunologyImmune Regulation +19

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

Bérangère Bertin, P. Véron, Christian Leborgne, +15

Scientific Reports

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, +12

Molecular Therapy — Methods & Clinical Development

Neonate Pompe MiceProvides Persistent EfficacyGene TherapiesImmunologyGene Vector +4

Gene Therapy in Patients with the Crigler–Najjar Syndrome

Lorenzo D’Antiga, Ulrich Beuers, Giuseppe Ronzitti, +16

New England Journal of Medicine

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler–Najjar Syndrome

Sem J. Aronson, P. Véron, Fanny Collaud, +14

Human Gene Therapy