- Genes & Diseases,Volume , Issue 2,2014, Pages 97-108
- Delivering on the promise of gene editing for cystic fibrosis
- Craig A.Hodges1, Ronald A.Conlon2
- 1.Department of Pediatrics, Case Western Reserve University, Cleveland, OH, USA;2.Department of Genetics and Genome Sciences, Case Western Reserve University, Cleveland, OH, USA
In this review, we describe a path for translation of gene editing into therapy for cystic fibrosis (CF). Cystic fibrosis results from mutations in the CFTR gene, with one allele predominant in patient populations. This simple, genetic etiology makes gene editing appealing for treatment of this disease. There already have been success in applying this approach to cystic fibrosis in cell and animal models, although these advances have been modest in comparison to advances for other disease.
CFTR geneCRISPR/Cas9Cystic fibrosisGene editingGene therapy
Copyright © 2014 Chongqing Medical University. Published by Elsevier B.V