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Amlexanox: Readthrough Induction and Nonsense-Mediated mRNA Decay Inhibition in a Charcot–Marie–Tooth Model of hiPSCs-Derived Neuronal Cells Harboring a Nonsense Mutation in GDAP1 Gene

Nonsense mutations are involved in multiple peripheral neuropathies. These mutations induce the presence of a premature termination codon (PTC) at the mRNA level. As a result, a dysfunctional or truncated protein is synthesized, or even absent linked to nonsense-mediated mRNA degradation (NMD) syste...

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Detalles Bibliográficos
Autores principales: Benslimane, Nesrine, Miressi, Federica, Loret, Camille, Richard, Laurence, Nizou, Angélique, Pyromali, Ioanna, Faye, Pierre-Antoine, Favreau, Frédéric, Lejeune, Fabrice, Lia, Anne-Sophie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10385573/
https://www.ncbi.nlm.nih.gov/pubmed/37513945
http://dx.doi.org/10.3390/ph16071034