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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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Bibliografski detalji
Izdano u:Haematologica
Glavni autori: Martorell, Lluis, Cortina, Vicente, Parra, Rafael, Barquinero, Jordi, Vidal, Francisco
Format: Artigo
Jezik:Inglês
Izdano: Ferrata Storti Foundation 2020
Teme:
Online pristup: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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