Prime editing in hematopoietic stem cells-From ex vivo to in vivo CRISPR-based treatment of blood disorders.

IF 4.9 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Jonas Holst Wolff, Jacob Giehm Mikkelsen
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引用次数: 2

Abstract

Prime editing of human hematopoietic stem cells has the potential to become a safe and efficient way of treating diseases of the blood directly in patients. By allowing site-targeted gene intervention without homology-directed repair donor templates and DNA double-stranded breaks, the invention of prime editing fuels the exploration of alternatives to conventional recombination-based ex vivo genome editing of hematopoietic stem cells. Prime editing is as close as we get today to a true genome editing drug that does not require a separate DNA donor. However, to adapt the technology to perform in vivo gene correction, key challenges remain to be solved, such as identifying effective prime editing guide RNAs for clinical targets as well as developing efficient vehicles to deliver prime editors to stem cells in vivo. In this review, we summarize the current progress in delivery of prime editors both in vitro and in vivo and discuss future challenges that need to be adressed to allow in vivo prime editing as a cure for blood disorders.

Abstract Image

Abstract Image

造血干细胞的启动编辑——从体外到体内基于crispr的血液疾病治疗
人类造血干细胞的初始编辑有可能成为直接治疗患者血液疾病的一种安全有效的方法。通过允许位点靶向基因干预,而无需同源定向修复供体模板和DNA双链断裂,先导编辑的发明推动了对传统的基于重组的造血干细胞体外基因组编辑的替代方法的探索。启动编辑是我们今天最接近真正的基因组编辑药物,不需要单独的DNA供体。然而,为了使这项技术在体内进行基因校正,仍有一些关键的挑战有待解决,比如为临床靶标识别有效的启动编辑指导rna,以及开发有效的载体将启动编辑器输送到体内干细胞。在这篇综述中,我们总结了目前在体外和体内递送引物编辑器的进展,并讨论了未来需要解决的挑战,以允许体内引物编辑作为血液疾病的治疗。
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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CiteScore
7.00
自引率
0.00%
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审稿时长
13 weeks
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