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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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Publicat a:J Clin Med
Autors principals: Borgatti, Monica, Altamura, Emiliano, Salvatori, Francesca, D’Aversa, Elisabetta, Altamura, Nicola
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2020
Matèries:
Accés en línia: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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