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相关概念视频

RNA Editing02:23

RNA Editing

9.7K
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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Long-patch Base Excision Repair01:02

Long-patch Base Excision Repair

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Since the discovery of the two BER pathways, there has been a debate about how a cell chooses one pathway over the other and the factors determining this selection. Numerous in vitro experiments have pointed out multiple determinants for the sub-pathway selection. These are:
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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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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...
1.6K
Base Excision Repair01:54

Base Excision Repair

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One of the common DNA damages is the chemical alteration of single bases by alkylation, oxidation, or deamination. The altered bases cause mispairing and strand breakage during replication. This type of damage causes minimal change to the DNA double helix structure and can be repaired by the base excision repair (BER) pathways. BER corrects damaged DNA sequences by removing the damaged base and restoring the original base sequence using the complementary strand as a template.
The first step of...
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Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

6.6K
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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相关实验视频

Updated: Jan 8, 2026

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

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工程基础编辑器通过定向进化减少了旁观者编辑.

Ramiro M Perrotta1, Svenja Vinke2, Raphaël Ferreira2,3

  • 1Department of Genetics, Harvard Medical School, Boston, MA, USA. Ramiro_perrotta@hms.harvard.edu.

Nature biotechnology
|December 18, 2025
PubMed
概括
此摘要是机器生成的。

研究人员设计了腺基编辑器 (ABE) 以提高基因组编辑精度. 这种新方法最大限度地减少了不必要的旁观者编辑,提高了治疗应用的安全性和效率.

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Functional Assessment of BRCA1 variants using CRISPR-Mediated Base Editors
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Last Updated: Jan 8, 2026

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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Functional Assessment of BRCA1 variants using CRISPR-Mediated Base Editors
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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科学领域:

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

背景情况:

  • 基准编辑器提供精确的基因组修改,但受到旁观者编辑的影响,限制了它们的使用.
  • 当前的精度策略往往会降低效率,并且是特定于序列的.

研究的目的:

  • 开发一个改进的基础编辑系统,尽量减少旁观者编辑,并保持高效率.
  • 建立一个可扩展的框架,用于设计高精度的基础编辑器.

主要方法:

  • 设计和测试3'-扩展导向RNA,以提高特异性.
  • 采用精确驱动的菌体辅助进化系统.
  • 利用蛋白质语言模型用于酶变体进化.

主要成果:

  • 确定了增强特异性的上下文依赖导向RNA变体.
  • 进化的腺基编辑器变体比ABE8e精确2-3倍.
  • 在体外各种病原性环境中实现了高的编辑效率.

结论:

  • 开发了一种平行工程方法,优化导向RNA和除氨酶酶.
  • 为基础编辑器精密工程创建了一个可扩展的框架.
  • 解决了基因组编辑的一个关键挑战,增强了治疗潜力.