Readthrough Activators and Nonsense-Mediated mRNA Decay Inhibitor Molecules: Real Potential in Many Genetic Diseases Harboring Premature Termination Codons

Readthrough Activators and Nonsense-Mediated mRNA Decay Inhibitor Molecules: Real Potential in Many Genetic Diseases Harboring Premature Termination Codons

Nonsense mutations that generate a premature termination codon (PTC) can induce both the accelerated degradation of mutated mRNA compared with the wild type version of the mRNA or the production of a truncated protein. One of the considered therapeutic strategies to bypass PTCs is their “readthrough...

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Bibliographic Details
Main Authors: Nesrine Benslimane, Camille Loret, Pauline Chazelas, Frédéric Favreau, Pierre-Antoine Faye, Fabrice Lejeune, Anne-Sophie Lia
Format: Article
Language:English
Published: MDPI AG 2024-02-01
Series:Pharmaceuticals
Subjects:
nonsense mutation
readthrough
premature termination codon (PTC)
genetic disease
nonsense-mediated mRNA decay (NMD)
translation
Online Access:https://www.mdpi.com/1424-8247/17/3/314

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