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Katharine Bushby et al, 2014, Muscle & Nerve CrossRef - Readthrough-Promoting Drugs Gentamicin and PTC124 Fail to Rescue Na v 1.5 Function of Human-Induced Pluripotent Stem Cell–Derived Cardiomyocytes Carrying Nonsense Mutations in the Sodium Channel Gene SCN5A
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Marina Y. Fosso et al, 2014, MedChemComm CrossRef - Synergy between Readthrough and Nonsense Mediated Decay Inhibition in a Murine Model of Cystic Fibrosis Nonsense Mutations
Daniel R. McHugh et al, 2020, International Journal of Molecular Sciences CrossRef - Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes
Florian Bolze et al, 2017, Scientific Reports CrossRef - Ataluren—Promising Therapeutic Premature Termination Codon Readthrough Frontrunner
Sylwia Michorowska, 2021, Pharmaceuticals CrossRef - Nonsense Suppression Therapy: New Hypothesis for the Treatment of Inherited Bone Marrow Failure Syndromes
Valentino Bezzerri et al, 2020, International Journal of Molecular Sciences CrossRef - Stop-codon and C-terminal nonsense mutations are associated with a lower risk of cardiac events in patients with long QT syndrome type 1
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Emma J. Brasell et al, 2019, PLOS ONE CrossRef - A Simple and Affordable Method to Create Nonsense Mutation Clones of p53 for Studying the Premature Termination Codon Readthrough Activity of PTC124
Chia-Chi Chen et al, 2023, Biomedicines CrossRef - Translational read-through of the RP2 Arg120stop mutation in patient iPSC-derived retinal pigment epithelium cells
Nele Schwarz et al, 2015, Human Molecular Genetics CrossRef - Absence of p.R50X Pygm read-through in McArdle disease cellular models
Guillermo Tarrasó et al, 2020, Disease Models & Mechanisms CrossRef - PTC124 Rescues Nonsense Mutation of Two Tumor Suppressor Genes NOTCH1 and FAT1 to Repress HNSCC Cell Proliferation
Ming-Han Wu et al, 2022, Biomedicines CrossRef - Functional Studies on Novel RET Mutations and Their Implications for Genetic Counseling for Hirschsprung Disease
Hui Wang et al, 2019, Frontiers in Genetics CrossRef - Ataluren‐driven restoration of Shwachman‐Bodian‐Diamond syndrome protein function in Shwachman‐Diamond syndrome bone marrow cells
Valentino Bezzerri et al, 2018, American Journal of Hematology CrossRef - Stop codons and the +4 nucleotide may influence the efficiency of G418 in rescuing nonsense mutations of the HERG gene
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