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Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids.
- Source :
-
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2016 Mar; Vol. 15 (2), pp. 158-62. Date of Electronic Publication: 2015 Aug 05. - Publication Year :
- 2016
-
Abstract
- 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 (E60X/4015delATTT, E60X/F508del, G542X/F508del, R1162X/F508del, W1282X/F508del and F508del/F508del). 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.<br /> (Copyright © 2015 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)
- Subjects :
- Cells, Cultured
Coccidiostats therapeutic use
Cystic Fibrosis genetics
Cystic Fibrosis metabolism
Cystic Fibrosis Transmembrane Conductance Regulator genetics
Cystic Fibrosis Transmembrane Conductance Regulator metabolism
Humans
RNA genetics
Codon, Nonsense drug effects
Cystic Fibrosis drug therapy
Cystic Fibrosis Transmembrane Conductance Regulator drug effects
Gentamicins therapeutic use
Organoids cytology
Oxadiazoles therapeutic use
Subjects
Details
- Language :
- English
- ISSN :
- 1873-5010
- Volume :
- 15
- Issue :
- 2
- Database :
- MEDLINE
- Journal :
- Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
- Publication Type :
- Academic Journal
- Accession number :
- 26255232
- Full Text :
- https://doi.org/10.1016/j.jcf.2015.07.007