Current avenues of gene therapy in Pompe disease

aDepartment of Pediatrics

bDepartment of Biochemistry & Molecular Biology, College of Medicine

cCenter for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL

dLafora Epilepsy Cure Initiative, USA

eDepartment of Physical Therapy, University of Florida, Gainesville, FL, United States

fBreathing Research and Therapeutics (BREATHE) Center, University of Florida, Gainesville, FL, USA

Correspondence to Carmen Leon-Astudillo, MD, 1600 SW Archer Road, Gainesville, FL 32608, USA. Tel: +1 352 273 8381; e-mail: [email protected]

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