Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
David Dwight Fuller, Sabhya Denise Rana, Prajwal Pradeep Thakre, Ethan S. Benevides, Megan K. Pope, Adrian Gary Todd, Victoria N. Jensen, Lauren A. Vaught, Denise A. Cloutier, Roberto A. Ribas, Reece C. Larson, Matthew S. Gentry, Ramon C. Sun, V. Ravi Chandran, Manuela Corti, , Barry J. Byrne
Molecular Therapy · Jun 14, 2025Peer-reviewedPhysiology