Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidaseFrancesco Puzzo, Federico Mingozzi, Dwight D. Koeberl et al.|Science Translational Medicine|2017Cited by 133
Exposure to wild-type AAV drives distinct capsid immunity profiles in humansKlaudia Kuranda, Federico Mingozzi, Priscilla Jean-Alphonse et al.|Journal of Clinical Investigation|2018Cited by 103
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE ExpressionPatrice Vidal, Federico Mingozzi, Serena Pagliarani et al.|Molecular Therapy|2017Cited by 37
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defectsPasqualina Colella, Federico Mingozzi, Pauline Sellier et al.|EBioMedicine|2020Cited by 27
Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-GlucosidaseUmut Cagin, Federico Mingozzi, Francesco Puzzo et al.|Molecular Therapy|2020Cited by 24