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 <i>GDAP1</i> 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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Principais autores:	Nesrine Benslimane, Federica Miressi, Camille Loret, Laurence Richard, Angélique Nizou, Ioanna Pyromali, Pierre-Antoine Faye, Frédéric Favreau, Fabrice Lejeune, Anne-Sophie Lia
Formato:	Artigo
Idioma:	Inglês
Publicado:	MDPI AG 2023-07-01
Series:	Pharmaceuticals
Assuntos:	GDAP1 CMT disease nonsense mutation nonsense-mediated decay (NMD) readthrough molecules premature termination codon (PTC)
Acceso en liña:	https://www.mdpi.com/1424-8247/16/7/1034
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