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Paper Title
Small-molecule eRF3a degraders rescue CFTR nonsense mutations by promoting premature termination codon readthrough.
PubMed
Paper Journal Title
Journal of Clinical Investigation
Paper Citation Count
9
Paper Publication Year
2022
Bio Mention
Bmi-1, Bmi-1/hTERT cell lines, CF, CF airway epithelial cell lines, CF transmembrane regulator, CFTR, CFTR nonsense mutations, CFTR variants, E3 ligase, ENaC, F508del-CFTR, F508del-CFTR cell lines, G418, G542X, G542X-CFTR, PTCs, W1282X, W1282X PTC variant, W1282X-CFTR, cell lines, cystic fibrosis, eRF3a, epithelial sodium channel, eukaryotic release factor 3a, hTERT, human, human TERT, mouse, mouse Bmi-1, parent primary cells, people, premature termination codon, premature termination codons, primary airway epithelial cells, primary cell, primary cells
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Author Name
Affiliation
Scott H Randell
Marsico Lung Institute and Cystic Fibrosis Research Center.
Scott H Randell
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