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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Detalhes bibliográficos
Principais autores: Craig A. Hodges, Ronald A. Conlon
Formato: Artigo
Idioma:English
Publicado em: KeAi Communications Co., Ltd. 2019-06-01
coleção:Genes and Diseases
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2352304218301363