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Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations

Rationale: Premature termination codons (PTCs) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (CF). Several agents are known to suppress PTCs but are poorly efficacious or toxic. Objectives: To determine whether there are clinically available agents that...

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Publicat a:Am J Respir Crit Care Med
Autors principals: Mutyam, Venkateshwar, Du, Ming, Xue, Xiaojiao, Keeling, Kim M., White, E. Lucile, Bostwick, J. Robert, Rasmussen, Lynn, Liu, Bo, Mazur, Marina, Hong, Jeong S., Falk Libby, Emily, Liang, Feng, Shang, Haibo, Mense, Martin, Suto, Mark J., Bedwell, David M., Rowe, Steven M.
Format: Artigo
Idioma:Inglês
Publicat: American Thoracic Society 2016
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5114449/
https://ncbi.nlm.nih.gov/pubmed/27104944
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1164/rccm.201601-0154OC
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