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- W4206439421 abstract "Objective: To investigate the regulatory effect of targeted siRNA on β-globin in erythroid cells cultured by targeted differentiation in vitro and provide new theoretical support for gene therapy for hemoglobin H (HbH) disease. Methods: Based on the β-globin gene expression results, the optimal siRNA sequence and its effective action dose were screened in erythroid cells, and the effect of the effective dose of the optimal siRNA on the regulation of β-globin expression and apoptosis in erythroid cells was examined. The effective dose of the optimal siRNA was applied to erythroid cells with HbH disease. The effects of transfected siRNAs on red line cells with HbH disease were comprehensively evaluated by measuring the expression of β-globin, reactive oxygen species (ROS) , and apoptosis rates. Results: Within 96 hours after transfection, siRNA2 significantly downregulated β-globin expression in in vitro cultured erythroid cells, but not α-globin. siRNA silencing effect and duration of effect were dose-dependent. siRNA2 downregulated β-globin expression, reduced intracellular ROS production, and decreased apoptosis rate in erythroid cells with HbH disease. Conclusion: Targeted siRNAs can downregulate β-globin expression, reduce intracellular ROS production, and downregulate apoptosis rate in erythroid cells with HbH disease.目的： 研究靶向siRNA对体外定向分化培养的红系细胞β-珠蛋白的调控作用，为血红蛋白H（Hemoglobin H, HbH）病的基因治疗提供新的理论支持。 方法： ①根据β-珠蛋白基因表达结果，在红系细胞中筛选出最佳siRNA序列及其有效作用剂量，检测有效剂量的最佳siRNA对红系细胞β-珠蛋白表达的调控和细胞凋亡的影响。②将有效剂量的最佳siRNA作用于HbH病红系细胞，检测转染后细胞β-珠蛋白表达、活性氧（ROS）和细胞凋亡率，综合评估转染siRNA对HbH病红系细胞的影响。 结果： ①siRNA(2)可在转染后96 h内显著下调体外培养的红系细胞β-珠蛋白的表达，但对α-珠蛋白无明显调控作用。siRNA(2)的沉默效应和效应持续时间均与作用剂量有关。②siRNA(2)可下调HbH病红系细胞β-珠蛋白表达，减少细胞内ROS生成，并减少细胞凋亡率。 结论： 靶向性siRNA可下调HbH病红系细胞β-珠蛋白表达，减少细胞内ROS产生，下调细胞凋亡率。." @default.
- W4206439421 created "2022-01-25" @default.
- W4206439421 creator A5007318688 @default.
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- W4206439421 date "2021-12-14" @default.
- W4206439421 modified "2023-09-27" @default.
- W4206439421 title "[Regulation effect of siRNA on β-globin in erythrocytes of hemoglobin H disease]." @default.
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- W4206439421 doi "https://doi.org/10.3760/cma.j.issn.0253-2727.2021.12.009" @default.
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