Article

Gene-Specific Nonsense-Mediated mRNA Decay Targeting for Cystic Fibrosis Therapy

2021-07-13

Abstract excerpt

Low CFTR mRNA expression due to nonsense-mediated mRNA decay (NMD) is a major hurdle in developing a therapy for cystic fibrosis (CF) caused by the W1282X mutation in the CFTR gene. CFTR-W1282X truncated protein retains partial function, so increasing its levels by inhibiting NMD of its mRNA will likely be beneficial. Because NMD regulates the normal expression of many genes, gene-specific stabilization of CFT...

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Literature Corpus work: 42244139-2def-59a5-b374-13f868a120a8
DOI: 10.1101/2021.07.13.452144

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