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Specific factor IX mRNA and protein features favor drug-induced readthrough over recurrent nonsense mutations
Drug-induced readthrough over premature stop codons (PTCs) is a potentially attractive therapy for genetic disorders, but a wide outcome variability has been observed. Through expression studies, we investigated the responsiveness to the readthrough-inducing drug geneticin of 11 rationally selected...
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| Publicat a: | Blood |
|---|---|
| Autors principals: | , , , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society of Hematology
2017
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5408560/ https://ncbi.nlm.nih.gov/pubmed/28196793 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2016-09-738641 |
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