Gene-Specific Nonsense-Mediated mRNA Decay Targeting for Cystic Fibrosis Therapy
Young Jin Kim, Tomoki T. Nomakuchi, Foteini Papaleonidopoulou, Lucia Yang, Qian Zhang, 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.
Nature Communications · May 27, 2022Peer-reviewedMolecular Biology