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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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| Publicado no: | Blood |
|---|---|
| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2017
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| Assuntos: | |
| Acesso em linha: | 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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