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相关概念视频

RNA Editing02:23

RNA Editing

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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...
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CRISPR01:59

CRISPR

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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...
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Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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Experimental RNAi02:15

Experimental RNAi

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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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Types of RNA01:23

Types of RNA

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Overview
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...
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CRISPR and crRNAs02:53

CRISPR and crRNAs

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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...
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相关实验视频

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A Nonsequencing Approach for the Rapid Detection of RNA Editing
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使用一种进化的细菌腺脱氨酶编程RNA编辑.

Hao Yan1,2, Weixin Tang3,4

  • 1Department of Chemistry, University of Chicago, Chicago, IL, USA.

Nature chemical biology
|July 5, 2024
PubMed
概括

研究人员开发了一种新的RNA编辑工具,称为DECOR (deaminase-enabled重编码RNA). 这种精确的遗传工具有效地纠正人类细胞中引起疾病的突变,显著减少了目标外影响.

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科学领域:

  • 分子生物学分子生物学
  • 基因工程是一种基因工程.
  • 生物技术是生物技术.

背景情况:

  • 编程RNA编辑是对遗传疾病的一种有前途的治疗方法.
  • 现有的RNA编辑平台面临着非目标效应的挑战.

研究的目的:

  • 开发一种具有高特异性和减少目标外影响的新型RNA编辑系统.
  • 在遗传疾病模型中展示这种新系统的治疗潜力.

主要方法:

  • 为可编程RNA编辑设计了一种大肠杆菌转移RNA腺脱氨酶 (TadA8e).
  • 利用CRISPR技术引导除氨酶到人类转录组中的特定位置.
  • 在各种类型的人类细胞中评估了目标和非目标编辑效率.
  • 在范德伍德综合征的小鼠模型中评估了系统的有效性.

主要成果:

  • 开发的系统,DECOR (脱氨酶激活的RNA重编码),在CRISPR指定的位置展示了高效的腺-氨酸编辑.
  • DECOR在目标上表现出与现有平台相似的活动,但其目标外影响减少了88%.
  • 一个高保真版本的DECOR进一步将非目标效应最小化到基底水平.
  • 通过DECOR介导的编辑成功地删除了IRF6转录中的病原性上游开放阅读框架,挽救了初级开放阅读框架表达.

结论:

  • DECOR代表了一种新且高度特定的RNA编辑技术.
  • 这种系统在遗传疾病中具有治疗应用的巨大潜力.
  • DECOR扩展了编程RNA编辑的工具包,并为遗传医学开辟了新的途径.