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相关概念视频

Conservative Site-specific Recombination and Phase Variation02:53

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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.
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CRISPR01:59

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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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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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相关实验视频

Updated: Jun 19, 2025

Using Sniper-Cas9 to Minimize Off-target Effects of CRISPR-Cas9 Without the Loss of On-target Activity Via Directed Evolution
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使用分子建模开发小型Cas9杂交物种.

Antoine Mangin1,2, Vincent Dion3,4, Georgina Menzies5

  • 1UK Dementia Research Institute at Cardiff University, Cardiff, CF24 4HQ, UK.

Scientific reports
|July 26, 2024
PubMed
概括
此摘要是机器生成的。

研究人员探索了较小的Cas9变体和混合体,以感染引起疾病的CAG/CTG重复,用于治疗神经肌肉疾病. 然而,这些工程系统未能实现基因编辑,这表明复杂的Cas9/sgRNA系统目前的in silico设计存在局限性.

关键词:
结合能量是有约束力的.这就是Cas9的情况.基因编辑 基因编辑分子动力学分子动力学

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CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy
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Efficient Production and Identification of CRISPR/Cas9-generated Gene Knockouts in the Model System Danio rerio
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相关实验视频

Last Updated: Jun 19, 2025

Using Sniper-Cas9 to Minimize Off-target Effects of CRISPR-Cas9 Without the Loss of On-target Activity Via Directed Evolution
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科学领域:

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

背景情况:

  • CAG/CTG重复收缩为15多种神经肌肉和神经退行性疾病提供治疗策略.
  • 目前使用Streptococcus pyogenes Cas9 (SpCas9) 的方法因酶大小而面临交付挑战,这阻碍了临床翻译.

研究的目的:

  • 为了研究较小的Cas9正态体 (SlugCas9,OgeuIscB) 和新型的Cas9/sgRNA杂交体,以有效地进行CAG/CTG重复收缩.
  • 为了克服在体内基因编辑应用中的腺相关病毒包装限制.

主要方法:

  • 选较小的Cas9正义体 (SlugCas9, OgeuIscB) 进行重复收缩活动.
  • 使用分子动力学和结合能量的计算,设计和合成Cas9/sgRNA混合系统.
  • 在人体细胞中Cas9/sgRNA杂交体内测试基因编辑效率.

主要成果:

  • SlugCas9 和 OgeuIscB 在诱导CAG/CTG重复收缩方面无效.
  • 开发的Cas9/sgRNA杂交对,尽管在形设计中,但未能编辑人类细胞中的目标序列.
  • 在的方法被证明不足以设计功能性的,大规模的Cas9/sgRNA复合体.

结论:

  • 较小的Cas9正规器件和设计的杂交器件不能有效地收缩CAG/CTG重复.
  • 目前的计算方法在设计治疗应用的复杂基因编辑系统方面是有限的.
  • 需要进一步的研究来开发可行的传递系统来治疗重复扩散疾病.