论文部分内容阅读
Purpose : In this essay, we explore whether CRISPR/CAS9 could correct the HBB mutation of β-Thal iPSCs.Methods: In this study, firstly, we correct the disease-causing mutations in bThal iPSCs through in situ gene targeting using the CRISPR/Cas9 system.After gene targeting, 18 clones were picked and expanded.They were then verified by PCR for HygR fragment integration into the genomic DNA.