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Literature Workspace

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1 reference

Research article 2023 Reviewed MVA Hackathon 2026 — BUB1B

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

Nesrine Benslimane, Federica Miressi, Camille Loret and 7 more Pharmaceuticals

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 synt… abstract via Crossref

Evidence snapshot note: Amlexanox in GDAP1 neuronal progenitors. Model: Human iPSC-derived neuronal progenitors with GDAP1 p.Ser194* (UGA). Where: Figures 2-4 · cited by MVA-AMLEXANOX-010

  • Cmt Disease
  • Nonsense Mutation
  • Premature Termination Codon (Ptc)
  • Nonsense-mediated Decay (Nmd)

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