Article
Recombinant Adeno-Associated Virus-Mediated Editing of the G551D Cystic Fibrosis Transmembrane Conductance Regulator Mutation in Ferret Airway Basal Cells.
Human gene therapy - 1 Oct 2022
Yan Ziying, Vorhies Kai, Feng Zehua, Park Soo Yeun, Choi Soon H, Zhang Yulong, Winter Michael, Sun Xingshen, Engelhardt John F
Abstract excerpt
Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (CF), a chronic disease that affects multiple organs, including the lung. We developed a CF ferret model of a scarless G551→D substitution in CFTR (CFTRG551D-KI), enabling approaches to correct this gating mutation in CF airways via gene editing. Homology-directed repair (HDR) was tested in Cas9-expressing CF...
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