Breakthrough RNA Therapy Shows Promise in Reversing ALS Nerve Damage

A recent study led by Eran Perlson at Tel Aviv University has revealed that a specific RNA molecule can halt and even reverse nerve cell damage caused by amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease. This advancement in mice could pave the way for innovative treatments against this fatal neurodegenerative condition.

ALS gradually paralyzes muscles by damaging neuromuscular junctions, the critical sites where nerve fibers connect with muscle cells. Until now, the mechanism triggering this damage remained unclear. Perlson’s research team previously observed that the toxic protein TDP-43 accumulates at these nerve-muscle junctions, impairing mitochondria—the energy-producing structures within nerve cells.

In healthy individuals, muscle cells produce microRNA-126, an RNA molecule that prevents TDP-43 buildup by being transported to nerve cells. However, the study published in Nature Neuroscience found that ALS patients exhibit decreased microRNA-126 production, contributing to nerve damage.

By restoring microRNA-126 levels in cultured ALS patient tissues and in affected mice, the researchers observed a reduction in toxic TDP-43 protein, decreased nerve cell death, and regeneration of damaged nerves. Perlson stated, "Our findings may serve as a foundation for developing effective gene therapies based on microRNA-126 supplementation, offering hope to millions affected by ALS worldwide."