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Testing of the therapeutic efficacy and safety of AMPA receptor RNA aptamers in an ALS mouse model RNA aptamers designed to block AMPA receptors effectively reduce progression of motor dysfunction, improve TDP-43 pathology, and prevent death of motor neurons in AR2 mice, a mouse model of sporadic ALS, without adverse effects
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Sánchez MB, Callaghan MJ, Selfe J, Twigg M, Smith T
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