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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Main Authors: | Nesrine Benslimane (Author), Federica Miressi (Author), Camille Loret (Author), Laurence Richard (Author), Angélique Nizou (Author), Ioanna Pyromali (Author), Pierre-Antoine Faye (Author), Frédéric Favreau (Author), Fabrice Lejeune (Author), Anne-Sophie Lia (Author) |
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Format: | Book |
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MDPI AG,
2023-07-01T00:00:00Z.
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