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

by Rémi Baroso, Pauline Sellier, Federica Defendi, Delphine Charignon, Arije Ghannam, Mohammed Habib, Christian Drouet, Bertrand Favier
Published 2016-01-01

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

by Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, Francesco Puzzo, Laetitia van Wittenberghe, Nicolas Guerchet, Nathalie Daniele, Bernard Gjata, Solenne Marmier, Severine Charles, Marcelo Simon Sola, Isabella Ragone, Christian Leborgne, Fanny Collaud, Federico Mingozzi
Published 2019-03-01

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

by Pasqualina Colella, Pauline Sellier, Manuel J. Gomez, Maria G. Biferi, Guillaume Tanniou, Nicolas Guerchet, Mathilde Cohen-Tannoudji, Maryse Moya-Nilges, Laetitia van Wittenberghe, Natalie Daniele, Bernard Gjata, Jacomina Krijnse-Locker, Fanny Collaud, Marcelo Simon-Sola, Severine Charles, Umut Cagin, Federico Mingozzi
Published 2020-11-01