Scientists develop microRNA-based gene therapy that halts ALS progression in mice

MedicalXpress | Jul 29, 2026

A single IV injection of a microRNA-based biologic developed by Guangping Gao, Ph.D., Robert H. Brown Jr., DPhil, MD, Jun Xie, Ph.D., and Zuoshang Xu, MD, Ph.D., suppressed production of the mutant SOD1 protein that causes amyotrophic lateral sclerosis (ALS), delayed disease onset by 60 days and extended lifespan by 100 days—more than tripling the average survival time—in mouse models of the disease.