Article
Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells.
Cell reports. Medicine - 21 May 2024
Bulcaen Mattijs, Kortleven Phéline, Liu Ronald B, Maule Giulia, Dreano Elise, Kelly Mairead, Ensinck Marjolein M, Thierie Sam, Smits Maxime, Ciciani Matteo, Hatton Aurelie, Chevalier Benoit, Ramalho Anabela S, Casadevall I Solvas Xavier, Debyser Zeger, Vermeulen François, Gijsbers Rik, Sermet-Gaudelus Isabelle, Cereseto Anna, Carlon Marianne S
Abstract excerpt
Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause cystic fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex...
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