遗传性血液病的基因治疗和基因编辑策略。

IF 6.6 2区 生物学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY
Xuemei Song, JinLei Liu, Tangcong Chen, Tingfeng Zheng, Xiaolong Wang, Xiang Guo
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引用次数: 0

摘要

基因疗法在治疗各种疾病,尤其是血友病、镰状细胞病和地中海贫血症等遗传性血液疾病方面显示出巨大的潜力。随着对疾病相关基因调控网络认识的不断深入,人们发现了更多的治疗靶点,尤其是β-血红蛋白病的治疗靶点。红细胞调控因子BCL11A为β-血红蛋白病提供了最有希望的治疗靶点,使用商业化基因治疗产品Casgevy减少其表达已于2023年在英国和美国获批使用。值得注意的是,创新基因编辑技术的出现进一步拓宽了基因治疗领域,为治疗提供了新的可能性。大量研究表明,以CRISPR技术为基础的碱基编辑和质粒编辑技术可以对造血干细胞进行精确的单碱基修饰,从而解决体内外遗传性血液疾病的问题。在这篇综述中,我们概述了基因疗法的现状,重点介绍了针对遗传性血液病的临床研究和基因治疗产品、潜在基因靶点的评估,以及目前基因治疗实践中采用的基因编辑工具,这为未来针对更广泛的疾病建立更安全、更有效的基因治疗方法提供了启示。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene therapy and gene editing strategies in inherited blood disorders.

Gene therapy has shown significant potential in treating various diseases, particularly inherited blood disorders such as hemophilia, sickle cell disease, and thalassemia. Advances in understanding the regulatory network of disease-associated genes have led to the identification of additional therapeutic targets for treatment, especially for β-hemoglobinopathies. Erythroid regulatory factor BCL11A offers the most promising therapeutic target for β-hemoglobinopathies and reduction of its expression using the commercialized gene therapy product Casgevy was approved for use in the UK and USA in 2023. Notably, the emergence of innovative gene editing technologies has further broadened the gene therapy landscape, presenting new possibilities for treatment. Intensive studies indicate that base editing and prime editing, built upon CRISPR technology, enable precise single-base modification in hematopoietic stem cells for addressing inherited blood disorders ex vivo and in vivo. In this review, we present an overview of the current landscape of gene therapies, focusing on clinical research and gene therapy products for inherited blood disorders, evaluation of potential gene targets, and the gene editing tools employed in current gene therapy practices, which provides an insight for the establishment of safer and more effective gene therapy methods for a wider range of diseases in the future.

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来源期刊
Journal of Genetics and Genomics
Journal of Genetics and Genomics 生物-生化与分子生物学
CiteScore
8.20
自引率
3.40%
发文量
4756
审稿时长
14 days
期刊介绍: The Journal of Genetics and Genomics (JGG, formerly known as Acta Genetica Sinica ) is an international journal publishing peer-reviewed articles of novel and significant discoveries in the fields of genetics and genomics. Topics of particular interest include but are not limited to molecular genetics, developmental genetics, cytogenetics, epigenetics, medical genetics, population and evolutionary genetics, genomics and functional genomics as well as bioinformatics and computational biology.
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