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

A azygous 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 accumulation of the mutant SOD1 macromolecule that causes amyotrophic lateral sclerosis (ALS), delayed illness onset by 60 days and extended lifespan by 100 days—more than tripling the mean endurance time—in rodent models of the disease.