2016-03-01
Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids
Publication
Publication
Journal of Cystic Fibrosis , Volume 15 - Issue 2 p. 158- 162
Premature termination codon read-through drugs offer opportunities for treatment of multiple rare genetic diseases including cystic fibrosis. We here analyzed the read-through efficacy of PTC124 and G418 using human cystic fibrosis intestinal organoids.
G418-mediated read-through induced only limited CFTR function, but functional restoration of CFTR by PTC124 could not be confirmed. These studies suggest that better read-through agents are needed for robust treatment of nonsense mutations in cystic fibrosis.
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| doi.org/10.1016/j.jcf.2015.07.007, hdl.handle.net/1765/82097 | |
| Journal of Cystic Fibrosis | |
| Organisation | Erasmus MC: University Medical Center Rotterdam |
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Zomer-van Ommen, D. D., Vijftigschild, L. A. W., Kruisselbrink, E., Vonk, A. M., Dekkers, J., Janssens, H., … Beekman, J. (2016). Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids. Journal of Cystic Fibrosis, 15(2), 158–162. doi:10.1016/j.jcf.2015.07.007 |
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