O.6Pre-clinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe diseaseSean M. Armour, Federico Mingozzi, Pauline Sellier et al.|Neuromuscular Disorders|2019Cited by 2
Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe diseaseSean M. Armour, Federico Mingozzi, Jayme M.L. Nordin et al.|Molecular Genetics and Metabolism|2020Cited by 0