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Variable readthrough responsiveness of nonsense mutations in hemophilia A

Readthrough therapy relies on the use of small molecules that enable premature termination codons in mRNA open reading frames to be misinterpreted by the translation machinery, thus allowing the generation of full-length, potentially functional proteins from mRNA carrying nonsense mutations. In pati...

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Detalles Bibliográficos
Autores principales:	Martorell, Lluis, Cortina, Vicente, Parra, Rafael, Barquinero, Jordi, Vidal, Francisco
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Ferrata Storti Foundation 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7012483/ https://www.ncbi.nlm.nih.gov/pubmed/31197069 http://dx.doi.org/10.3324/haematol.2018.212118

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