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Gene Therapy in Combination with Nitrogen Scavenger Pretreatment Corrects Biochemical and Behavioral Abnormalities of Infant Citrullinemia Type 1 Mice by Andrea Bazo, Aquilino Lantero, Itsaso Mauleón, Leire Neri, Martin Poms, Johannes Häberle, Ana Ricobaraza, Bernard Bénichou, Jean-Philippe Combal, Gloria Gonzalez-Aseguinolaza, Rafael Aldabe
Published 2022-11-01
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Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy by Nicholas D. Weber, Leticia Odriozola, Irene Ros-Gañán, Guillermo García-Porrero, David Salas, Josepmaria Argemi, Jean-Philippe Combal, Takashi K. Kishimoto, Gloria González-Aseguinolaza
Published 2023-05-01
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Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy by Emmanuel Gonzales, Antoine Gardin, Marion Almes, Amaria Darmellah-Remil, Hanh Seguin, Charlotte Mussini, Stéphanie Franchi-Abella, Mathieu Duché, Oanez Ackermann, Alice Thébaut, Dalila Habes, Bogdan Hermeziu, Martine Lapalus, Thomas Falguières, Jean-Philippe Combal, Bernard Benichou, Sonia Valero, Anne Davit-Spraul, Emmanuel Jacquemin
Published 2023-10-01
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