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
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
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patientsElisa Masat, Yann Péréon, Pascal Laforêt et al.|Scientific Reports|2016Cited by 32
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
O.6Pre-clinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe diseaseSean M. Armour, Federico Mingozzi, Jayme M.L. Nordin et al.|Neuromuscular Disorders|2019Cited by 2