Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy

Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy

The W1282X nonsense mutation in the CFTR gene causes cystic fibrosis by reducing its mRNA and functional protein levels. Here the authors developed antisense-oligonucleotide cocktails that restore CFTR protein function by gene-specific stabilization of CFTR mRNA.

Bibliographic Details
Main Authors: Young Jin Kim, Tomoki Nomakuchi, Foteini Papaleonidopoulou, Lucia Yang, Qian Zhang, Adrian R. Krainer
Format: Article
Language:English
Published: Nature Portfolio 2022-05-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-30668-y

Internet

https://doi.org/10.1038/s41467-022-30668-y

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