Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor

New drugs are needed to enhance premature termination codon (PTC) suppression to treat the underlying cause of cystic fibrosis (CF) and other diseases caused by nonsense mutations. We tested new synthetic aminoglycoside derivatives expressly developed for PTC suppression in a series of complementary...

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Hlavní autoři: Xue, Xiaojiao, Mutyam, Venkateshwar, Tang, Liping, Biswas, Silpak, Du, Ming, Jackson, Laura A., Dai, Yanying, Belakhov, Valery, Shalev, Moran, Chen, Fuquan, Schacht, Jochen, J. Bridges, Robert, Baasov, Timor, Hong, Jeong, Bedwell, David M., Rowe, Steven M.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Thoracic Society 2014
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Original Research
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4068923/
https://ncbi.nlm.nih.gov/pubmed/24251786
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1165/rcmb.2013-0282OC
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