Long-term correction of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integration
Xi Chen, Xuran Niu, Yang Liu, Rui Zheng, Lei Yang, Jian Lu, Shuming Yin, Wei Yu, Jiahao Pan, Ahmed M. Sayed, Xueyun Ma, Meizhen Liu, Fengxiang Jing, Mingyao Liu, Jiazhi Hu, Liren Wang, Dali Li
Topics & Concepts
CRISPRFactor IXLocus (genetics)Cas9Homologous chromosomeBiologyHomology (biology)Homologous recombinationGeneticsComputational biologyClotting factorGeneMolecular biologyMedicineInternal medicineCRISPR and Genetic EngineeringVirus-based gene therapy researchPluripotent Stem Cells Research