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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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Haematologica
Päätekijät: Martorell, Lluis, Cortina, Vicente, Parra, Rafael, Barquinero, Jordi, Vidal, Francisco
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Ferrata Storti Foundation 2020
Aiheet:
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC7012483/
https://ncbi.nlm.nih.gov/pubmed/31197069
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2018.212118
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