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Targeted pseudouridylation: An approach for suppressing nonsense mutations in disease genes
Affiliation:1. Department of Biochemistry and Biophysics, Center for RNA Biology, University of Rochester Medical Center, Rochester, NY, USA;2. Center for Biomedical Informatics and Wilmot Cancer Institute, University of Rochester Medical Center, Rochester, NY, USA;3. ProQR Therapeutics, Leiden, the Netherlands
Abstract:
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  • Keywords:RNA modification  pseudouridine  nonsense mutations  nonsense suppression  premature termination codon  nonsense-mediated mRNA decay  box H/ACA RNA  targeted pseudouridylation  PTC read-through
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