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胎儿血红蛋白再生治疗β-血红蛋白病

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  • 华东师范大学 生命科学学院,上海市基因编辑和细胞治疗前沿科学研究基地,上海市调控生物学重点实验室, 上海 200241

收稿日期: 2021-11-12

  网络出版日期: 2022-07-18

Fetal hemoglobin regeneration for therapy of β-hemoglobinopathies

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  • Shanghai Key Laboratory of Regulatory Biology, Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, School of Life Sciences, East China Normal University, Shanghai 200241, China

Received date: 2021-11-12

  Online published: 2022-07-18

摘要

 β-血红蛋白病是世界上最常见的遗传疾病,该病最初的治疗策略为病毒载体介导的基因治疗,但由于花费昂贵、疗效有限、存在安全隐患等问题进展缓慢。近些年,基因组编辑技术成为开发β-血红蛋白病新型治愈方案的有力工具。文章回顾了胎儿血红蛋白再生策略治疗β-血红蛋白病的最新进展,其中破坏BCL11A红系增强子的方式因其安全、有效、临床价值高而备受青睐。β-血红蛋白病基因治疗策略的下一步发展值得期待,有望完全治愈β-地中海贫血和镰状细胞贫血症这两种遗传性血液病。

本文引用格式

赵飞燕, 吴宇轩 . 胎儿血红蛋白再生治疗β-血红蛋白病[J]. 自然杂志, 2022 , 44(5) : 389 -397 . DOI: 10.3969/j.issn.0253-9608.2022.03.011

Abstract

β-hemoglobinopathy is the most common genetic disease in the world. The initial treatment strategy is viral vector-mediated gene therapy. Due to the high cost, limited efficacy and potential safety problems, the progress is slow. In recent years, genome editing technology has been a powerful tool for the development of new cures for β-hemoglobinopathy. This article reviewed the latest developments in fetal hemoglobin regeneration strategies for the treatment of β-hemoglobinopathy. The stratergy of disrupting the BCL11A erythroid enhancer is favored due to its safety, effectiveness and higher clinical value. Looking forward to development of gene therapy strategy for β-hemoglobinopathy, it is expected to completely cure the two inherited blood diseases of β-thalassemia and sickle cell anemia. 
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