Article

Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing

2024-08-29

Abstract excerpt

<h4>Summary</h4> Cystic Fibrosis (CF) is a life-shortening autosomal recessive disease caused by mutations in the CFTR gene, resulting in functional impairment of the encoded ion channel. F508del mutation, a trinucleotide deletion, is the most frequent cause of CF affecting approximately 80% of patients. Even though current pharmacological treatments alleviate the F508del-CF disease symptoms there is no definiti...

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Literature Corpus work: 40e03de3-6c68-5baf-aecb-e2d9c4ac7c6f
DOI: 10.1101/2024.08.28.610115

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