Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids

February 17, 2022

Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids

J Cyst Fibros | May 2021

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G970R-CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect

February 16, 2022

G970R-CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect

Clin Transl Sci | March 2021

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Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation

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

February 16, 2022

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

Cell Rep | February 2019

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