Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations

Sharon J. Brown, Rafael J. Yáñez-Muñoz, Heidi R. Fuller

Neural Regeneration Research . 2025, (7): 2011 -2012 . DOI: 10.4103/NRR.NRR-D-24-00504