Gene Therapy Shows Promise in Halting ALS Progression in Mice

Scientists at UMass Chan Medical School have developed a microRNA-based gene therapy that successfully halted the progression of Amyotrophic Lateral Sclerosis (ALS) in mouse models. A single intravenous injection preserved motor neurons, maintained neuromuscular connections, and significantly improved muscle and respiratory function, motor performance, and lifespan. These findings, published in Nature Communications, show unprecedented therapeutic benefits and hold potential for clinical application in SOD1-caused ALS and other neurodegenerative diseases.