可编程的表观基因组编辑通过短暂的CRISPR表观基因组编辑蛋白
Da Xu1, Swen Besselink1,2, Gokul N Ramadoss3
1Department of Molecular and Cell Biology, University of California, Berkeley, CA, USA.
Nature communications
|August 26, 2025
概括
我们开发了一种名为RENDER的新方法, 将表观基因组编辑器输入人体细胞. 这种系统可以使基因沉默和抑制与疾病相关的蛋白质,促进研究和治疗的表观基因组编辑.
科学领域:
- 分子生物学
- 基因编辑
- 表观遗传学
背景情况:
- 可编程的表观基因组编辑器改变基因表达而不破坏DNA.
- 基于CRISPR的编辑器很大,限制了它们的应用.
- 对研究和治疗而言,表观基因组编辑器的有效交付至关重要.
研究的目的:
- 为表观基因组编辑器开发一个新的交付平台.
- 为了实现抑制剂和激活剂的暂时输送.
- 克服目前基于CRISPR的系统的尺寸限制.
主要方法:
- 开发出强大的表观基因组编辑核蛋白 (RENDER).
- 工程核蛋白复合物用于短暂的输送.
- 应用RENDER来提供CRISPRi,DNMT3A-3L-dCas9,CRISPRoff和TET1-dCas9.
主要成果:
- 在不同类型的人类细胞,包括T细胞中,RENDER实现了内源基因的长期表观遗传沉默.
- 在人类干细胞衍生的神经元中成功抑制了目标基因.
- 与神经退行性疾病相关的V337M突变Tau蛋白的表达减少.
结论:
- RENDER是一个先进的平台, 可以将表观基因组编辑器输入人体细胞.
- 这项技术扩大了基因组编辑在基础研究中的范围.
- RENDER具有未来治疗应用的潜力.
更多相关视频
相关概念视频
CRISPR/Cas9 Genome Editing
213
The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
213
CRISPR
52.9K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
52.9K
CRISPR and crRNAs
17.4K
Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
17.4K


