CRISPR for cystic fibrosis: Advances and insights from a systematic review.

Abstract excerpt

Cystic fibrosis (CF) is a severe genetic disorder caused by loss-of-function mutations in the CFTR gene. Gene-editing approaches have the potential to correct such mutations. This systematic review outlines the mechanisms of the main CRISPR-based technologies, and, through cross-study comparisons, analyzes 27 research articles that applied them to target CF-causing variants. We report and discuss the strategy...

Topics

• Cystic Fibrosis
• Humans
• Gene Editing
• Cystic Fibrosis Transmembrane Conductance Regulator
• CRISPR-Cas Systems
• Genetic Therapy