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Nonaminoglycoside compounds induce readthrough of nonsense mutations
Journal of Experimental Medicine, 10/06/09
Du L et al. – Two leading compounds consistently induced functional ATM protein in ATM–deficient cells containing disease–causing nonsense mutations, as demonstrated by direct measurement of ATM protein, restored ATM kinase activity, and colony survival assays for cellular radiosensitivity. The two compounds also demonstrated readthrough activity in mdx mouse myotube cells carrying a nonsense mutation and induced significant amounts of dystrophin protein.
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