For more information: Cell Research Authors: Mo Li, Keiichiro Suzuki, Jing Qu, Preeti Saini, Ilir Dubova, Fei Yi, Jungmin Lee, Ignacio Sancho - Martinez, Guang - Hui Liu and Juan Carlos Izpisua Belmonte Efficient correction of hemoglobinopathy - causing
mutations by homologous recombination in integration - free patient iPSCs
An independent team led by Hans Clevers at Hubrecht Institute in The Netherlands used the CRISPR / Cas9 genome editing system to correct the cystic fibrosis transmembrane conductor receptor (CFTR)
by homologous recombination in cultured intestinal stem cells of patients with cystic fibrosis.
In brief, the entire ORF of the RhoH gene located in the third exon was replaced with a cassette consisting of a lacZ and neomycin resistance gene (LacZ / neo)
by homologous recombination.
A, Targeting vector was designed to disrupt the RhoH gene
by homologous recombination.