Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives.

Abstract

Premature stop codons are the result of nonsense mutations occurring within the coding sequence of a gene. These mutations lead to the synthesis of a truncated protein and are responsible for several genetic diseases. A potential pharmacological approach to treat these diseases is to promote the translational readthrough of premature stop codons by small… (More)
DOI: 10.1016/j.ejmech.2015.06.038

Topics

7 Figures and Tables

Cite this paper

@article{Pibiri2015EnhancementOP, title={Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives.}, author={Ivana Pibiri and Laura Lentini and Raffaella Melfi and Giulia Gallucci and Andrea Pace and Angelo Spinello and Giampaolo Barone and Aldo Di Leonardo}, journal={European journal of medicinal chemistry}, year={2015}, volume={101}, pages={236-44} }