Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
Article in Molecular Metabolism (February 2024)
The most recent citing publications are shown below. View all 21 publications that cite this research output on Dimensions.
Muscle diffusion MRI reveals autophagic buildup in a mouse model for Pompe disease
Article in Scientific Reports (December 2023)
From Acid Alpha-Glucosidase Deficiency to Autophagy: Understanding the Bases of POMPE Disease
Article in International Journal of Molecular Sciences (August 2023)