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, Laetitia van Wittenberghe 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
Functional, biochemical and transcriptional rescue of advanced Pompe disease in mice with liver expression of secretable GAAUmut Cagin, Federico Mingozzi, Francesco Puzzo et al.|Molecular Genetics and Metabolism|2019Cited by 0
Liver expression of secretable GAA rescues advanced Pompe disease at the biochemical, functional, and transcriptional level in Gaa miceUmut Cagin, Federico Mingozzi, Francesco Puzzo et al.|Molecular Genetics and Metabolism|2020Cited by 0