Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 32243806.
- Also identified by DOI 10.1016/j.stem.2020.03.012.
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Abstract
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.
Medical subject headings
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator