Efficient Gene Editing at Major CFTR Mutation Loci

• Main Authors: Jinxue Ruan (Author), Hiroyuki Hirai (Author), Dongshan Yang (Author), Linyuan Ma (Author), Xia Hou (Author), Hong Jiang (Author), Hongguang Wei (Author), Carthic Rajagopalan (Author), Hongmei Mou (Author), Guoshun Wang (Author), Jifeng Zhang (Author), Kui Li (Author), Yuqing E. Chen (Author), Fei Sun (Author), Jie Xu (Author)
• Format: Book
• Published: Elsevier, 2019-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...