New ALS therapy

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

July 29, 2026
9:51 pm

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.

The gene therapy, delivered via an adeno-associated virus (AAV) vector, preserved motor neurons and maintained neuromuscular connections in treated animals. This translated into improved muscle and respiratory function, motor performance and lifespan in preclinical studies. These findings, published in Nature Communications, have the potential for clinical application in patients with SOD1-caused ALS, as well as other neurodegenerative diseases caused by toxic, gain-of-function gene mutations.

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

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