Molecular BiologyNature CommunicationsPeer-reviewed
Gene-Specific Nonsense-Mediated mRNA Decay Targeting for Cystic Fibrosis Therapy
Young Jin Kim, Tomoki T. Nomakuchi, Foteini Papaleonidopoulou…·Adrian R. Krainer
This study develops a cocktail of ASOs to specifically increase CFTR W1282X mRNA and protein levels by inhibiting NMD, improving chloride current in human bronchial epithelial cells.
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