Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice
Jun Xie
Nature Communications, Published online: 23 May 2026; doi:10.1038/s41467-026-73545-8 In mice, a next-generation gene therapy for spinal muscular atrophy improved survival and motor function more than a Zolgensma-like treatment, with fewer signs of liver and heart toxicity, supporting its promise as a safer, more effective approach.