造血干细胞和祖细胞基因编辑:超越血液病。

IF 4.9 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Frontiers in genome editing Pub Date : 2022-01-01 DOI:10.3389/fgeed.2022.997142
Valentina Buffa, José Roberto Alvarez Vargas, Anne Galy, Simone Spinozzi, Céline J Rocca
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引用次数: 2

摘要

从造血干细胞和祖细胞(HSPCs)移植治疗严重遗传性疾病或癌症的数十年实践中吸取的经验教训,为目前使用自体基因修饰的造血干细胞和祖细胞进行体外基因治疗奠定了基础,迄今已治疗了数百名单基因疾病患者。随着对造血干细胞和祖细胞生物学知识的增加,患者适应方式的改进以及新的基因编辑技术的出现,一个基于造血干细胞和祖细胞的基因治疗的新时代即将出现。基因编辑有可能恢复突变基因的生理表达,或者将功能基因插入精确的位点,减少脱靶活性和毒性。患者条件的进步降低了治疗毒性,并可能改善基因修饰细胞和特定子代的植入。由于这些改进,各种血液或免疫疾病以及其他遗传性疾病的新的潜在治疗方法将继续出现。在本综述中,将报道造血干细胞和祖细胞基因编辑的最新进展,重点关注这种方法如何成为治疗非血液相关遗传性疾病的有希望的解决方案,并讨论治疗作用背后的机制。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders.

Lessons learned from decades-long practice in the transplantation of hematopoietic stem and progenitor cells (HSPCs) to treat severe inherited disorders or cancer, have set the stage for the current ex vivo gene therapies using autologous gene-modified hematopoietic stem and progenitor cells that have treated so far, hundreds of patients with monogenic disorders. With increased knowledge of hematopoietic stem and progenitor cell biology, improved modalities for patient conditioning and with the emergence of new gene editing technologies, a new era of hematopoietic stem and progenitor cell-based gene therapies is poised to emerge. Gene editing has the potential to restore physiological expression of a mutated gene, or to insert a functional gene in a precise locus with reduced off-target activity and toxicity. Advances in patient conditioning has reduced treatment toxicities and may improve the engraftment of gene-modified cells and specific progeny. Thanks to these improvements, new potential treatments of various blood- or immune disorders as well as other inherited diseases will continue to emerge. In the present review, the most recent advances in hematopoietic stem and progenitor cell gene editing will be reported, with a focus on how this approach could be a promising solution to treat non-blood-related inherited disorders and the mechanisms behind the therapeutic actions discussed.

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CiteScore
7.00
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0.00%
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审稿时长
13 weeks
期刊最新文献
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