Article

Elucidation of Global Trends in the Effects of VX-661 and VX-445 on the Expression of Clinical CFTR Variants

2022-10-15

Abstract excerpt

Cystic fibrosis (CF) is a chronic genetic disease caused by mutations that compromise the expression and/ or function of the cystic fibrosis transmembrane conductance regulator chloride channel (CFTR). Most people with CF harbor a common misfolded CFTR variant (ΔF508), which can be rescued by combination therapies containing “corrector” compounds that restore its expression. Nevertheless, there are over 400 other...

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Literature Corpus work: 791aa20b-2360-5496-9973-a48b2121e276
DOI: 10.1101/2022.10.14.512300

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