舞台上的精确表观基因组编辑:一种调节基因表达的新方法。

IF 3.2 Q2 GENETICS & HEREDITY Epigenetics Insights Pub Date : 2018-12-13 eCollection Date: 2018-01-01 DOI:10.1177/2516865718818838
Claudio Mussolino
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引用次数: 7

摘要

在过去的几十年里,对人类病理的更好理解揭示了遗传改变和表观遗传畸变可以是疾病的驱动因素或加剧其表现。可定制平台的可用性允许精确的基因组靶向,通过直接解决疾病的起源,开辟了治愈遗传疾病的可能性。事实上,将不同的效应物拴在dna结合片段上,可以实现基因组、转录组或表观基因组的精确改变,从而使致病畸变正常化。设计核酸酶用于治疗性基因组编辑目前正在接近临床,并且出现了关于脱靶效应的安全问题。表观基因组编辑可能是一个有价值的替代方案,因为它不依赖于DNA双链断裂(最有害的DNA损伤形式之一)来发挥其功能。我们最近描述了设计师表观基因组修饰物(DEM),这是一种在临床相关的原代人类细胞中实现精确表观基因组编辑的新平台。我们讨论了DEM的效率,并强调了它们显著的安全性,这当然使这个平台成为未来临床翻译的有价值的候选者。
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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Precise Epigenome Editing on the Stage: A Novel Approach to Modulate Gene Expression.

In the last decades, a better understanding of human pathologies has revealed that genetic alterations as well as epigenetic aberrations can be drivers of a disease or exacerbate its manifestation. The availability of customizable platforms that allow precise genomic targeting has opened the possibility to cure genetic disorders by tackling directly the origin of the disease. Indeed, tethering of different effectors to a DNA-binding moiety grants precise alterations of the genome, transcriptome, or epigenome with the aim of normalizing disease-causing aberrations. The use of designer nucleases for therapeutic genome editing is currently approaching the clinics, and safety concerns arise with respect to off-target effects. Epigenome editing might be a valuable alternative, as it does not rely on DNA double-strand breaks, one of the most deleterious form of DNA damage, to exert its function. We have recently described designer epigenome modifier (DEM), a novel platform for achieving precise epigenome editing in clinically relevant primary human cells. We discuss the efficiency of DEM and highlight their remarkable safety profile, which certainly makes this platform a valuable candidate for future clinical translation.

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来源期刊
Epigenetics Insights
Epigenetics Insights GENETICS & HEREDITY-
CiteScore
5.10
自引率
0.00%
发文量
10
审稿时长
8 weeks
期刊最新文献
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