I did not expect it but there was a huge blizzard I was dropped down a little bit further from the base, it was snowing Our Intelligence told us they didn't get the news of Nemesis being destroyed, the Colonel said we could get critical Information to win the war so I agreed. Apparently radio was really bad there so most communication there is down. All rights reserved.They fixed the Nemesis Holon and I was transferred into it, I was given an undercover mission In summary, our study provides a new approach which can be used for the treatment of β-hemoglobinopathies in the future.īCL11A erythroid Enhancer CRISPR/Cas9 Multiplex gene editing β-hemoglobinopathies γ-globin promoter.Ĭopyright © 2022 Elsevier B.V. No off-target mutations were detected, suggesting its accuracy in HSPCs. Amplicon deep sequencing and whole genome sequencing were used to detect the editing frequency of on- and potential off-target sites in CD34 + cells. By further optimizing the on-target DNA editing efficiency of multiplex gene editing, the percentage of F-cells exceeded 50% in HUDEP-2 cells. ![]() Multiplex gene editing led to higher γ-globin expression than single-gene editing without inhibiting erythroid differentiation. Here, we performed single-gene editing and multiplex gene editing via CRISPR/Cas9 technology to edit BCL11A erythroid-specific enhancer and BCL11A binding site on γ-globin gene promoter in HUDEP-2 cells and adult human CD34 + cells. BCL11A is an important transcription factor that suppresses the γ-globin expression, which makes it one of the most promising therapeutic targets in β-hemoglobinopathies. One strategy to cure this disease relies on re-activating the γ-globin expression. Electronic address: are caused by mutations in the β-globin gene. Electronic address: 8 School of Medicine, Guizhou University, Guiyang, 550025, China Prenatal Diagnosis Center, Guizhou Provincial People's Hospital, Guiyang, 550002, China NHC Key Laboratory of Pulmonary Immunological Diseases, Guizhou Provincial People's Hospital, Guiyang, 550002, China. Electronic address: 7 CAS Key Laboratory of Genome Science & Information, Beijing Institute of Genomics, Chinese Academy of Sciences/ China National Center for Bioinformation, Beijing, 100101, China University of Chinese Academy of Sciences, Beijing, 100049, China Institute for Stem Cell and Regeneration, Chinese Academy of Sciences, Beijing, 100101, China.
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