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A G542X cystic fibrosis mouse model for examining nonsense mutation directed therapies

Nonsense mutations are present in 10% of patients with CF, produce a premature termination codon in CFTR mRNA causing early termination of translation, and lead to lack of CFTR function. There are no currently available animal models which contain a nonsense mutation in the endogenous Cftr locus tha...

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Bibliografische gegevens
Gepubliceerd in:PLoS One
Hoofdauteurs: McHugh, Daniel R., Steele, Miarasa S., Valerio, Dana M., Miron, Alexander, Mann, Rachel J., LePage, David F., Conlon, Ronald A., Cotton, Calvin U., Drumm, Mitchell L., Hodges, Craig A.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Public Library of Science 2018
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6010256/
https://ncbi.nlm.nih.gov/pubmed/29924856
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0199573
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