Nature Communications (Apr 2022)
CFTR mRNAs with nonsense codons are degraded by the SMG6-mediated endonucleolytic decay pathway
Abstract
Currently, there is no therapy for patients with cystic fibrosis caused by nonsense mutations. Here the authors show that CFTR mRNAs with nonsense codons are predominantly degraded by the SMG6-mediated branch of the NMD pathway, providing potential therapeutic strategies for the devastating disease.