Efficient Gene Editing at Major CFTR Mutation Loci

Efficient Gene Editing at Major CFTR Mutation Loci

Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Nuclease-mediated precise gene editing (PGE) represents a promising therapy for CF, for which an efficient strategy that is free of viral vector, d...

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Bibliographic Details
Main Authors: Jinxue Ruan, Hiroyuki Hirai, Dongshan Yang, Linyuan Ma, Xia Hou, Hong Jiang, Hongguang Wei, Carthic Rajagopalan, Hongmei Mou, Guoshun Wang, Jifeng Zhang, Kui Li, Yuqing E. Chen, Fei Sun, Jie Xu
Format: Article
Language:English
Published: Elsevier 2019-06-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253119300368

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http://www.sciencedirect.com/science/article/pii/S2162253119300368

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