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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
Autores principales: Borgatti, Monica, Altamura, Emiliano, Salvatori, Francesca, D’Aversa, Elisabetta, Altamura, Nicola
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7073686/
https://www.ncbi.nlm.nih.gov/pubmed/31972957
http://dx.doi.org/10.3390/jcm9020289