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

• Main Authors: Nesrine Benslimane (Author), Camille Loret (Author), Pauline Chazelas (Author), Frédéric Favreau (Author), Pierre-Antoine Faye (Author), Fabrice Lejeune (Author), Anne-Sophie Lia (Author)
• Format: Book
• Published: MDPI AG, 2024-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 "readth...