Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids
J Cyst Fibros | May 2021
February 16, 2022
G970R-CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect
Clin Transl Sci | March 2021
February 16, 2022
Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation
J Cyst Fibros | Sep 2021 | Clinical trial
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Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis
Cell Rep | February 2019
