Nonsense codons suppression. An acute toxicity study of three optimized TRIDs in murine model, safety and tolerability evaluation

Nonsense codons suppression. An acute toxicity study of three optimized TRIDs in murine model, safety and tolerability evaluation

Stop mutations cause 11% of the genetic diseases, due to the introduction of a premature termination codon (PTC) in the mRNA, followed by the production of a truncated protein. A promising therapeutic approach is the suppression therapy by Translational Readthrough Inducing Drugs (TRIDs), restoring...

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Bibliographic Details
Main Authors: Federica Corrao, Maria Grazia Zizzo, Marco Tutone, Raffaella Melfi, Ignazio Fiduccia, Pietro Salvatore Carollo, Aldo Di Leonardo, Gaetano Caldara, Riccardo Perriera, Andrea Pace, Beatrice Belmonte, Selene Sammataro, Ivana Pibiri, Laura Lentini
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Biomedicine & Pharmacotherapy
Subjects:
Nonsense mutation
Premature termination codon (PTC)
Oxadiazoles
Translational readthrough inducing drugs (TRIDs)
Toxicity study
Online Access:http://www.sciencedirect.com/science/article/pii/S0753332222012756

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http://www.sciencedirect.com/science/article/pii/S0753332222012756

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