Article
Screening Readthrough Compounds to Suppress Nonsense Mutations: Possible Application to β-Thalassemia
21 Jan 2020
Abstract excerpt
Several types of thalassemia (including β039-thalassemia) are caused by nonsense mutations in genes controlling globin production, leading to premature translation termination and mRNA destabilization mediated by the nonsense mediated mRNA decay. Drugs (for instance, aminoglycosides) can be designed to suppress premature translation termination by inducing readthrough (or nonsense suppression) at the premature...
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