A CRISPR view of hematopoietic stem cells: Moving innovative bioengineering into the clinic.
A CRISPR view of hematopoietic stem cells: Moving innovative bioengineering into the clinic.
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DOI：
10.1002/ajh.26588
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发表时间：
2022-09
影响因子：
12.8
通讯作者：
中科院分区：
文献类型：
--
作者：
成簇规则间隔短回文重复序列 (CRISPR)/Cas 基因组工程已成为一种强大的工具，可以以无与伦比的准确性和效率修改精确的基因组序列。过去 5 年 CRISPR 技术的重大进步推动了造血研究新技术的发展，以探究造血干细胞 (HSC) 生物学的复杂性。特别是，使用各种 Cas“风味”以及测序和/或功能输出的基于 CRISPR 的高通量筛选正变得越来越高效和易于使用。在这篇综述中，我们讨论了 CRISPR 介导的基因组工程的最新成就，以及这些新工具如何促进对 HSC 异质性和整个生命周期功能的理解。此外，我们还重点介绍了如何使用这些技术来回答以前无法解决的问题以及实施这些技术的挑战。最后，我们关注它们在临床模型和治疗血液疾病方面的转化潜力。 CRISPR/Cas 基因组工程的出现推动了造血研究的发展，提高了我们对造血干细胞生物学的理解。这些进步已迅速转化为基于 CRISPR/Cas 的新型疗法，改善了血液疾病的临床治疗方式。
Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome engineering has emerged as a powerful tool to modify precise genomic sequences with unparalleled accuracy and efficiency. Major advances in CRISPR technologies over the last 5 years have fueled the development of novel techniques in hematopoiesis research to interrogate the complexities of hematopoietic stem cell (HSC) biology. In particular, high throughput CRISPR based screens using various “flavors” of Cas coupled with sequencing and/or functional outputs are becoming increasingly efficient and accessible. In this review, we discuss recent achievements in CRISPR‐mediated genomic engineering and how these new tools have advanced the understanding of HSC heterogeneity and function throughout life. Additionally, we highlight how these techniques can be used to answer previously inaccessible questions and the challenges to implement them. Finally, we focus on their translational potential to both model and treat hematological diseases in the clinic. The advent of CRISPR/Cas genome engineering has pushed the envelope of hematopoiesis research at the bench, improving our understanding of hematopoietic stem cell biology. The advances made have rapidly translated to novel CRISPR/Cas based therapies, improving how hematological diseases are treated in the clinic.
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影响因子：
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作者：
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通讯作者：
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影响因子：
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影响因子：
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作者：
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