Delivering on the promise of gene editing for cystic fibrosis

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 dise...

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Bibliographic Details
Main Authors: Craig A. Hodges, Ronald A. Conlon
Format: Article
Language:English
Published: KeAi Communications Co., Ltd. 2019-06-01
Series:Genes and Diseases
Online Access:http://www.sciencedirect.com/science/article/pii/S2352304218301363