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相关概念视频

CRISPR01:59

CRISPR

51.1K
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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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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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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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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可编程RNA基编辑与可光激活的CRISPR-Cas13

Jeonghye Yu1, Jongpil Shin1, Jihwan Yu1

  • 1Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea.

Nature communications
|January 22, 2024
PubMed
概括

研究人员开发了一种可感应光的CRISPR-Cas13系统 (paCas13) 用于RNA操纵. 该系统能够精确控制细胞和动物模型中的RNA降解和基编辑,提供新的治疗途径.

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科学领域:

  • 分子生物学分子生物学
  • 基因编辑技术的技术
  • 生物技术是生物技术.

背景情况:

  • CRISPR-Cas13系统是用于RNA操纵的多功能工具,包括干扰,成像和编辑.
  • 现有的CRISPR-Cas13系统缺乏对其活动的精确时间和空间控制.
  • 诱导系统的开发对于精确的生物研究和治疗应用至关重要.

研究的目的:

  • 开发一种可感应光的CRISPR-Cas13系统,用于对RNA调制的时空控制.
  • 为可逆RNA编辑创建一个光感应基编辑器.
  • 为了证明这些新型RNA调节系统的体外和体内适用性.

主要方法:

  • 开发与Magnet融合的分裂-Cas13系统 (paCas13),确定最佳的N351/C350分裂地点.
  • 将ADAR2与催化不活的paCas13片段融合,以创建一个可诱导光的基编辑器 (padCas13).
  • 在哺乳动物细胞和小鼠模型中测试paCas13和padCas13系统的RNA扰动,基编辑 (A-to-I,C-to-U) 和转录调制.

主要成果:

  • 该paCas13系统显示了高诱导性和低背景活性,用于光诱导的RNA扰动.
  • 在光控制下,padCas13编辑器启用了可逆RNA基编辑 (A-to-I,C-to-U) 在体外.
  • 在padCas13编辑器成功地调整了翻译后的修改,并在小鼠模型中激活了目标转录 in vivo.

结论:

  • 一个新的光感应CRISPR-Cas13系统 (paCas13) 和一个基编辑器 (padCas13) 成功开发.
  • 这些系统提供精确的,光线依赖的控制RNA降解和基编辑在体外和体内.
  • paCas13系统在各种疾病状态和生理过程中对RNA操纵具有广泛的适用性,促进了研究和治疗开发.