Novel Gene Therapy Shows Unprecedented Success in Halting ALS Progression in Mice

Scientists at UMass Chan Medical School developed a microRNA-based gene therapy significantly suppressing the mutant SOD1 protein causing ALS in mice. A single injection delayed disease onset by 60 days and extended lifespan by 100 days, tripling average survival. Published in Nature Communications, these "unprecedented" findings offer substantial hope for clinical application in SOD1-caused ALS and other neurodegenerative conditions, marking a major step.