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U7 small nuclear RNA splice-switching therapeutics for STMN2 and UNC13A in Amyotrophic Lateral Sclerosis

Puja R Mehta1,2, Tomas Solomon3,2, Sarah Pickles4,2

  • 1Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.

Biorxiv : the Preprint Server for Biology
|December 15, 2025
PubMed

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