Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing.

Abstract excerpt

Since the early days of its conceptualization and application, human gene transfer held the promise of a permanent solution to genetic diseases including cystic fibrosis (CF). This field went through alternated periods of enthusiasm and distrust. The development of refined technologies allowing site specific modification with programmable nucleases highly revived the gene therapy field. CRISPR nucleases and...

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• CRISPR-Cas Systems
• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator
• Disease Models, Animal
• Gene Editing