Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo

摘要

Gene correction presents one of few options for a cure for all patients with cystic fibrosis. This commentary discusses new applications of CRISPR-based gene editing technology with increased efficiency and specificity to correct the cystic fibrosis transmembrane regulator (CFTR) function in patient-specific primary epithelial cells.