Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons

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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Bibliographic Details
Main Authors: Iwona Pranke, Laure Bidou, Natacha Martin, Sandra Blanchet, Aurélie Hatton, Sabrina Karri, David Cornu, Bruno Costes, Benoit Chevalier, Danielle Tondelier, Emmanuelle Girodon, Matthieu Coupet, Aleksander Edelman, Pascale Fanen, Olivier Namy, Isabelle Sermet-Gaudelus, Alexandre Hinzpeter
Format: Article
Language:English
Published: European Respiratory Society 2018-02-01
Series:ERJ Open Research
Online Access:http://openres.ersjournals.com/content/4/1/00080-2017.full

Internet

http://openres.ersjournals.com/content/4/1/00080-2017.full

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