RNA Boost Reawakens Nerves in ALS Lab Models, Tel Aviv Team Says
Researchers at Tel Aviv University say they have identified a molecular switch in amyotrophic lateral sclerosis (ALS) and used RNA-based gene therapy to halt—and even reverse—nerve damage in lab models, offering a potential path to treatment for a disease long considered incurable. The international team’s study, led in Israel by Prof. Eran Perlson with collaborators at Sheba Medical Center, the Weizmann Institute, Ben-Gurion University, and partners in France, Turkey, and Italy, was published in Nature Neuroscience. Their approach targets how and why motor neurons fail at the neuromuscular junction, the meeting point of nerve and muscle.
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Perlson’s group focused on toxic clumps of the protein TDP-43 that build up where nerve fibers meet muscle. The team found that healthy muscle sends tiny packets carrying microRNA-126 across the synapse to prevent excess TDP-43 from forming. In ALS, that protective signal drops. “When we added a specific RNA molecule to human cells and animal models for ALS, the nerve cells stopped degenerating and even regenerated,” the researchers reported. Prof. Perlson said the work “gets to the root” of junction failure that precedes paralysis: “Most patients die within three to five years of diagnosis, due to paralysis of the diaphragm muscles and respiratory failure.”
Lead author Dr. Ariel Ionescu explained the mechanism: reduced microRNA-126 allows TDP-43 to aggregate and damage mitochondria, starving neurons of energy. Restoring microRNA-126 in patient-derived tissues and ALS mice lowered TDP-43, protected the junction, and spurred regrowth. “Adding microRNA-126 rescues neurons damaged by ALS,” the team concluded, framing the method as a gene-therapy candidate. Perlson called the discovery “a critical molecular mechanism of ALS in its early stages,” and said it could underpin drugs aimed at stabilizing the junction before widespread motor neuron loss.
