Strategies against Nonsense: Oxadiazoles as Translational Readthrough-Inducing Drugs (TRIDs).
ataluren
cystic fibrosis
nonsense mutation
oxadiazoles
premature termination codon
translational readthrough inducing drugs
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
06 Jul 2019
06 Jul 2019
Historique:
received:
14
06
2019
revised:
01
07
2019
accepted:
02
07
2019
entrez:
10
7
2019
pubmed:
10
7
2019
medline:
18
12
2019
Statut:
epublish
Résumé
This review focuses on the use of oxadiazoles as translational readthrough-inducing drugs (TRIDs) to rescue the functional full-length protein expression in mendelian genetic diseases caused by nonsense mutations. These mutations in specific genes generate premature termination codons (PTCs) responsible for the translation of truncated proteins. After a brief introduction on nonsense mutations and their pathological effects, the features of various classes of TRIDs will be described discussing differences or similarities in their mechanisms of action. Strategies to correct the PTCs will be presented, particularly focusing on a new class of Ataluren-like oxadiazole derivatives in comparison to aminoglycosides. Additionally, recent results on the efficiency of new candidate TRIDs in restoring the production of the cystic fibrosis transmembrane regulator (CFTR) protein will be presented. Finally, a prospectus on complementary strategies to enhance the effect of TRIDs will be illustrated together with a conclusive paragraph about perspectives, opportunities, and caveats in developing small molecules as TRIDs.
Identifiants
pubmed: 31284579
pii: ijms20133329
doi: 10.3390/ijms20133329
pmc: PMC6651739
pii:
doi:
Substances chimiques
Codon, Nonsense
0
Oxadiazoles
0
Pharmaceutical Preparations
0
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Fondazione per la Ricerca sulla Fibrosi Cistica
ID : FFC#3/2017
Organisme : Fondazione 3° Pilastro Internazionale
ID : 2018
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