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Breakthrough RNA Therapy Halts ALS Progression in Trials

11/19/2025

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Summary

Story summary

Tel Aviv University researchers have developed RNA-based gene therapy that halts deterioration in ALS.
The study identifies microRNA-126, which stops neuron degeneration and promotes regeneration in ALS models.
The research involved collaboration with France, Turkey, and Italy.
ALS patients produce less microRNA-126, leading to TAR DNA-binding protein 43 (TDP-43) accumulation that harms motor neurons.
The findings point to a new therapeutic avenue for ALS, with plans for clinical trials.

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Story summary

Tel Aviv University researchers have developed RNA-based gene therapy that halts deterioration in ALS.
The study identifies microRNA-126, which stops neuron degeneration and promotes regeneration in ALS models.
The research involved collaboration with France, Turkey, and Italy.
ALS patients produce less microRNA-126, leading to TAR DNA-binding protein 43 (TDP-43) accumulation that harms motor neurons.
The findings point to a new therapeutic avenue for ALS, with plans for clinical trials.