Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidaseFrancesco Puzzo, Federico Mingozzi, Pasqualina Colella 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
Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance InductionSang-oh Han, Dwight D. Koeberl, Giuseppe Ronzitti et al.|Molecular Therapy — Methods & Clinical Development|2017Cited by 77
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