Article
CRISPR/Cas9 (D10A) nickase-mediated Hb CS gene editing and genetically modified fibroblast identification.
Bioengineered - 1 May 2022
Wu Wei-Hao, Ma Xiao-Mei, Huang Jian-Qing, Lai Qin, Jiang Fu-Neng, Zou Cui-Yun, Chen Long-Tian, Yu Lian
Abstract excerpt
This study investigated whether CRISPR/Cas9 (D10A) nickase-mediated gene editing can correct the aberrant Hb Constant Spring mutation (Hb CS or HBA2: c.427 T > C) in fibroblasts. Vectors for repairing the α-globin-encoding gene, HBA2:c.427 T > C mutation, includingthe CRISPR/Cas9(D10A)-sg plasmid and donor with homology arms, were constructed and used to perform gene editing in patient-derived fibroblasts. We...
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