A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion
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DOI: 10.1038/s41467-021-24575-x
Abstract: Premature termination codons (PTCs) prevent translation of a full-length protein and trigger nonsense-mediated mRNA decay (NMD). Nonsense suppression (also termed readthrough) therapy restores protein function by selectively suppressing translation termination at PTCs. Poor efficacy of… read more here.
Keywords: readthrough; translational readthrough; small molecule; induces translational ... See more keywords