相关实验视频

Updated: Sep 17, 2025

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Published on: March 29, 2019

在体内无载体的呼吸道内,活体表观遗传编辑

Naohiro Yano¹, Mohankumar Ramar¹, David J Gregory¹

¹Medical School of Brown University, Division of Surgical Research, Department of Surgery, Rhode Island Hospital, NAB B001. 593 Eddy Street, Providence, RI 02903, USA.

Trends in biotechnology

|July 3, 2025

概括

直接注射的表观遗传编辑蛋白消除了DNA甲基化,使得在没有遗传修饰的情况下能够在体内有针对性的基因表达调节. 这为治疗基因控制提供了一个有希望的,可翻译的方法.

科学领域:

表观遗传学和基因调控
分子生物学分子生物学
生物技术是生物技术.

背景情况:

删除DNA甲基化是一种控制基因表达的关键策略.
目前的方法通常依赖于转基因表达,限制了临床翻译.
对于表观遗传编辑而言,存在对非病毒性,直接传递方法的需求.

研究的目的:

开发和验证基于蛋白质的重组表观遗传编辑器,用于直接在体内给药.
为了实现有针对性的,促进体特定的DNA脱甲基化,而无需转基因或病毒载体.
为了证明有针对性的脱甲基化对基因表达的功能后果.

主要方法:

工程重组蛋白质包含dCas9或指域用于促进体向.
纳入蒂胺-DNA-糖酶 (TDG) 和十一转位 (Tet) 催化域进行脱甲基化.
在实验室和体内直接给小鼠肺细胞注射蛋白质;分析核内输送,DNA脱甲基化和基因表达变化.

主要成果:

在体外和体内证明成功输送表观遗传编辑蛋白的核内输送.
在特定的促进体中实现了向的DNA脱甲基化.
观察到基因特异性的转录性脱抑,导致功能性细胞反应,如干扰素敏感性.

结论:

在体内无载体,有针对性的促进体去甲基化的原理证明,使用直接注射的蛋白质编辑器.
这种方法绕过了转基因或病毒传递的需要,增强了治疗潜力.
突出了精确表观遗传修饰和基因表达控制的新策略.

关键词:

Cxcl1111 在线阅读气溶喷雾剂是一种气溶.脱甲基化脱甲基化编辑表观遗传学编辑聚变脱甲基酶的融合在空中通道内的空中通道.肺动脉肺动脉是什么意思没有载体的无载体.

更多相关视频

In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing

In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing

Published on: May 5, 2023

CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery

CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery

Published on: May 30, 2025

相关概念视频

Epigenetic Regulation

Epigenetic Regulation

Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.

Position-effect Variegation

Position-effect Variegation

In 1928, a German botanist Emil Heitz observed the moss nuclei with a DNA binding dye. He observed that while some chromatin regions decondense and spread out in the interphase nucleus, others do not. He termed them euchromatin and heterochromatin, respectively. He proposed that the heterochromatin regions reflect a functionally inactive state of the genome. It was later confirmed that heterochromatin is transcriptionally repressed, and euchromatin is transcriptionally active chromatin.

RNA Editing

RNA Editing

RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...

Spreading of Chromatin Modifications

Spreading of Chromatin Modifications

The histone proteins in the nucleosomes are post-translationally modified (PTM) to increase or decrease access to DNA. The commonly observed PTMs are methylation, acetylation, phosphorylation, and ubiquitination of lysine amino acids in the histone H3 tail region. These histone modifications have specific meaning for the cell. Hence, they are called "histone code". The protein complex involved in histone modification is termed as "reader-writer" complex.
Writers
The writer...