CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cells

Objective To edit the γ-globin promoter of HEK293T cells using the CRISPR/Cas9 technology to upregulate γ-globin and explore the feasibility of using gene editing technology for the treatment of β-thalassemia. Methods Using gene editing technology to treat β-thalassemia, two sgRNA sequences were des...

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Main Authors: HUANG Xianjuan, LAI Yongrong, LI Jing
Format: Article
Language:zho
Published: Editorial Office of Journal of Guangxi Medical University 2025-04-01
Series:Guangxi Yike Daxue xuebao
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Online Access:https://journal.gxmu.edu.cn/article/doi/10.16190/j.cnki.45-1211/r.2025.02.011
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author HUANG Xianjuan
LAI Yongrong
LI Jing
author_facet HUANG Xianjuan
LAI Yongrong
LI Jing
author_sort HUANG Xianjuan
collection DOAJ
description Objective To edit the γ-globin promoter of HEK293T cells using the CRISPR/Cas9 technology to upregulate γ-globin and explore the feasibility of using gene editing technology for the treatment of β-thalassemia. Methods Using gene editing technology to treat β-thalassemia, two sgRNA sequences were designed at 420 bp upstream of the γ -globin transcription start site (TSS). CRISPR/Cas9 plasmids were transfected via lipofectamine to induce cleavage of the hemoglobin gamma (HBG) gene promoter in HEK293T cells. Positive clone screening, Sanger sequencing validation, TIDE analysis for editing efficiency, reverse transcription-quantitative polymerase chain reaction (RT-qPCR) and western blotting were used to detect the expression level of the γ-globin gene. Results Both sgRNAs achieved efficient gene cutting on γ-globin, with cutting of 44.20% (sgRNA1) and 32.90% (sgRNA2), respectively. The results of RT-qPCR and western blotting showed that, compared to the negative control groups (NCs transfected with empty PX459 plasmid), the expression levels of the γ-globin gene in the sgRNA2 group were elevated (P < 0.05). Conclusion The CRISPR/Cas9 gene editing technology can achieve effective editing on the γ-globin promoter, thereby upregulating γ-globin.
format Article
id doaj-art-e931ed1ad720437c8474dbe258da810f
institution OA Journals
issn 1005-930X
language zho
publishDate 2025-04-01
publisher Editorial Office of Journal of Guangxi Medical University
record_format Article
series Guangxi Yike Daxue xuebao
spelling doaj-art-e931ed1ad720437c8474dbe258da810f2025-08-20T02:15:20ZzhoEditorial Office of Journal of Guangxi Medical UniversityGuangxi Yike Daxue xuebao1005-930X2025-04-0142224124610.16190/j.cnki.45-1211/r.2025.02.011gxykdxxb-42-2-241CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cellsHUANG Xianjuan0LAI Yongrong1LI Jing2The First Affiliated Hospital of Guangxi Medical University, Nan‐ ning 530021, ChinaThe First Affiliated Hospital of Guangxi Medical University, Nan‐ ning 530021, ChinaThe First Affiliated Hospital of Guangxi Medical University, Nan‐ ning 530021, ChinaObjective To edit the γ-globin promoter of HEK293T cells using the CRISPR/Cas9 technology to upregulate γ-globin and explore the feasibility of using gene editing technology for the treatment of β-thalassemia. Methods Using gene editing technology to treat β-thalassemia, two sgRNA sequences were designed at 420 bp upstream of the γ -globin transcription start site (TSS). CRISPR/Cas9 plasmids were transfected via lipofectamine to induce cleavage of the hemoglobin gamma (HBG) gene promoter in HEK293T cells. Positive clone screening, Sanger sequencing validation, TIDE analysis for editing efficiency, reverse transcription-quantitative polymerase chain reaction (RT-qPCR) and western blotting were used to detect the expression level of the γ-globin gene. Results Both sgRNAs achieved efficient gene cutting on γ-globin, with cutting of 44.20% (sgRNA1) and 32.90% (sgRNA2), respectively. The results of RT-qPCR and western blotting showed that, compared to the negative control groups (NCs transfected with empty PX459 plasmid), the expression levels of the γ-globin gene in the sgRNA2 group were elevated (P < 0.05). Conclusion The CRISPR/Cas9 gene editing technology can achieve effective editing on the γ-globin promoter, thereby upregulating γ-globin.https://journal.gxmu.edu.cn/article/doi/10.16190/j.cnki.45-1211/r.2025.02.011crispr/cas9 editingγ-globinhek293t cellsgene editing
spellingShingle HUANG Xianjuan
LAI Yongrong
LI Jing
CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cells
Guangxi Yike Daxue xuebao
crispr/cas9 editing
γ-globin
hek293t cells
gene editing
title CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cells
title_full CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cells
title_fullStr CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cells
title_full_unstemmed CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cells
title_short CRISPR/Cas9-mediated γ-globin expression upregulation in HEK293T cells
title_sort crispr cas9 mediated γ globin expression upregulation in hek293t cells
topic crispr/cas9 editing
γ-globin
hek293t cells
gene editing
url https://journal.gxmu.edu.cn/article/doi/10.16190/j.cnki.45-1211/r.2025.02.011
work_keys_str_mv AT huangxianjuan crisprcas9mediatedgglobinexpressionupregulationinhek293tcells
AT laiyongrong crisprcas9mediatedgglobinexpressionupregulationinhek293tcells
AT lijing crisprcas9mediatedgglobinexpressionupregulationinhek293tcells