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Screening Readthrough Compounds to Suppress Nonsense Mutations: Possible Application to β-Thalassemia

Several types of thalassemia (including β(0)39-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 design...

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Detalles Bibliográficos
Publicado en:J Clin Med
Main Authors: Borgatti, Monica, Altamura, Emiliano, Salvatori, Francesca, D’Aversa, Elisabetta, Altamura, Nicola
Formato: Artigo
Idioma:Inglês
Publicado: MDPI 2020
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7073686/
https://ncbi.nlm.nih.gov/pubmed/31972957
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm9020289
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