Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidaseFrancesco Puzzo, Federico Mingozzi, Laetitia van Wittenberghe et al.|Science Translational Medicine|2017Cited by 133
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
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
Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenesPasqualina Colella, Federico Mingozzi, Francesco Puzzo et al.|Neuromuscular Disorders|2017Cited by 1