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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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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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...
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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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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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RNA Interference01:23

RNA Interference

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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
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Experimental RNAi02:15

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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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相关实验视频

Updated: Jul 16, 2025

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

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精确的RNA基编辑使用工程和内源效应器.

Laura S Pfeiffer1, Thorsten Stafforst2,3

  • 1Interfaculty Institute of Biochemistry, University of Tübingen, Tübingen, Germany.

Nature biotechnology
|September 21, 2023
PubMed
概括
此摘要是机器生成的。

RNA基编辑精确地改变RNA分子中的遗传信息,用于治疗应用. 本综述涵盖了策略,重点关注作用于RNA (ADAR) 酶的腺胺酶,以提高效率和体内输送.

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Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.
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科学领域:

  • 分子生物学分子生物学
  • 生物技术是生物技术.
  • 遗传学 是一个遗传学.

背景情况:

  • RNA基编辑会在RNA分子内重写遗传信息.
  • 人体细胞中的酶催化核基转化,使RNA重编码成为可能.
  • 与DNA编辑不同,RNA编辑是可逆和可剂量的,具有治疗潜力.

研究的目的:

  • 审查当前的局部导向RNA基编辑策略.
  • 为了突出编辑效率,精度和交付方面的进步.
  • 讨论利用内源性腺脱氨酶作用于RNA (ADAR) 酶.

主要方法:

  • 总结当前的局部导向RNA基编辑策略.
  • 审查工程编辑效应器和内源ADAR酶策略.
  • 分析改进编辑效率,精度和体内传输方面的成就.

主要成果:

  • 目前的RNA基编辑侧重于腺和细胞脱胺.
  • 最近的进展提高了编辑效率,精度和编码准范围.
  • 在体内输送到与疾病相关的组织是一个关键的重点.

结论:

  • 基RNA编辑提供可逆和可剂量的治疗应用.
  • 该领域预计将很快推出用于遗传疾病的第一个RNA基编辑药物.
  • 未来的挑战包括优化RNA基编辑以实现生物过程的安全和可剂量调制.