Pauline Sellier

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

Francesco Puzzo, Pasqualina Colella, Maria Grazia Biferi et al. · Science Translational Medicine · 2017 · 129 citations · Full text

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 et al. · Molecular Therapy — Methods & Clinical Development · 2018 · 70 citations · Full text

Neonate Pompe Mice, Provides Persistent Efficacy, Gene Therapies +6

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates

Helena Costa Verdera, Fanny Collaud, Christopher Riling et al. · Nature Communications · 2021 · 36 citations · Full text

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

Pasqualina Colella, Pauline Sellier, Manuel J. Gómez et al. · EBioMedicine · 2020 · 27 citations · Full text

Kininogen Cleavage Assay: Diagnostic Assistance for Kinin-Mediated Angioedema Conditions

Rémi Baroso, Pauline Sellier, Federica Defendi et al. · PLoS ONE · 2016 · 23 citations · Full text