Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons

Premature termination codons (PTCs) are generally associated with severe forms of genetic diseases. Readthrough of in-frame PTCs using small molecules is a promising therapeutic approach. Nonetheless, the outcome of preclinical studies has been low and variable. Treatment efficacy depends on: 1) the...

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Detalhes bibliográficos
Publicado no:ERJ Open Res
Main Authors: Pranke, Iwona, Bidou, Laure, Martin, Natacha, Blanchet, Sandra, Hatton, Aurélie, Karri, Sabrina, Cornu, David, Costes, Bruno, Chevalier, Benoit, Tondelier, Danielle, Girodon, Emmanuelle, Coupet, Matthieu, Edelman, Aleksander, Fanen, Pascale, Namy, Olivier, Sermet-Gaudelus, Isabelle, Hinzpeter, Alexandre
Formato: Artigo
Idioma:Inglês
Publicado em: European Respiratory Society 2018
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5827411/
https://ncbi.nlm.nih.gov/pubmed/29497617
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1183/23120541.00080-2017
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