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Molecular Therapy : the Journal of the American Society of Gene Therapy|March 8, 2021
Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase ComplexGustavo de Alencastro, Francesco Puzzo, Mara Pavel-Dinu, et al.Scientific Reports|November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patientsElisa Masat, Pascal Laforêt, Marie De Antonio, et al.Hepatology (Baltimore, Md.)|March 21, 2017
Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectorsMarianna Hösel, Anke Huber, Susanne Bohlen, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 12, 2020
Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-GlucosidaseUmut Cagin, Francesco Puzzo, Manuel Jose Gomez, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|February 4, 2018
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE ExpressionPatrice Vidal, Serena Pagliarani, Pasqualina Colella, et al.Science Translational Medicine|December 1, 2017
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidaseFrancesco Puzzo, Pasqualina Colella, Maria G Biferi, et al.Pageof 2