Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations
1 School of Pharmacy and Bioengineering, Keele University, Keele, UK (Brown SJ, Fuller HR).
2 Wolfson Center for Inherited Neuromuscular Disease, TORCH Building, RJAH Orthopaedic Hospital, Oswestry, UK (Brown SJ, Fuller HR).
3 AGCTlab.org, Center of Gene and Cell Therapy, Department of Biological Sciences, School of Life Sciences and the Environment, Royal Holloway University of London, Egham, UK (Yáñez-Muñoz RJ).