MEDiscuss • Medical News • Antisense therapy for spinal muscular atrophy shows promise
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Antisense therapy for spinal muscular atrophy shows promise
Scientists have succeeded in reversing symptoms of type III SMA, a relatively mild form of spinal muscular atrophy, in mice by introducing chemically modified RNA pieces called ASOs into their spinal cords. The ASOs fix the molecular mistake underlying SMA by redirecting a cellular editing process called alternative splicing.
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