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可编程的表观基因组编辑通过暂时传递CRISPR表观基因组编辑 ribonucleoproteins

Da Xu, Swen Besselink, Gokul N Ramadoss

bioRxiv : the preprint server for biology

|December 9, 2024

概括

一种名为RENDER的新方法使表观基因组编辑器能够短暂地传递到人体细胞中. 这种方法克服了基于CRISPR的工具的尺寸限制,为研究和潜在疗法提供基因表达调制的便利.

科学领域:

分子生物学分子生物学
表观遗传学在表观遗传学中,表观遗传学是指表观遗传学.
基因编辑技术的技术

背景情况:

可编程的表观基因组编辑器通过修改染色质而改变基因表达,而不会破坏DNA.
基于CRISPR的表观基因组编辑器面临着由于其庞大尺寸的交付挑战,限制了它们在研究和治疗中的应用.
有效的交付系统对于推进表观基因组编辑应用至关重要.

研究的目的:

开发一种新型的交付平台,在人类细胞中暂时引入可编程表观基因组编辑器.
为了克服与传统基于CRISPR的表观基因组编辑系统相关的尺寸限制.
证明新平台在调节基因表达的有效性及其潜在的治疗应用.

主要方法:

开发一个强大的ENveloped交付表观基因组编辑器核蛋白蛋白 (RENDER) 平台.
提供含有表观遗传抑制剂 (CRISPRi,DNMT3A-3L-dCas9,CRISPRoff) 和激活剂 (TET1-dCas9) 的核蛋白复合体.
在各种人体细胞类型中应用,包括初级T细胞和干细胞衍生神经元.

主要成果:

染器使表观基因组编辑器作为核糖蛋白复合体的暂时传递成为可能.
在不同的人类细胞类型中证明了内源基因的持久表观遗传沉默.
成功应用RENDER来抑制与神经退行性疾病相关的Tau蛋白在人类干细胞衍生的神经元中.

结论:

RENDER平台提供了一种有效的方法,用于将基于CRISPR的表观基因组编辑器输入人体细胞.
RENDER克服了尺寸限制,扩大了基本研究中表观基因组编辑的范围.
这项技术对未来的治疗应用具有前景,包括神经退行性疾病.

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