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相关概念视频

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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CRISPR and crRNAs02:53

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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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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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工程金属有机框架用于有针对性的CRISPR/Cas9基因编辑

Navid Rabiee1,2,3,4, Mohammad Rabiee5

  • 1Department of Basic Medical Science, School of Medicine, Tsinghua University, Beijing 100084, China.

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概括

工程金属有机框架 (MOF) 为CRISPR/Cas9基因编辑提供了先进的交付,提高了精度和效率. 这些生物相容的纳米平台显示出在精密医学中更安全,更有针对性的基因治疗的前景.

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科学领域:

  • 纳米技术 纳米技术
  • 生物医学工程 生物医学工程
  • 分子生物学分子生物学

背景情况:

  • 对于治疗用途,CRISPR/Cas9基因编辑需要高效和精确的输送系统.
  • 非病毒载体对于安全有效的基因传递至关重要,但往往面临局限性.
  • 金属有机框架 (MOFs) 是一种具有可调节性质的新型无机纳米载体.

研究的目的:

  • 审查用于CRISPR/Cas9交付的基于MOF的纳米平台的设计和应用.
  • 突出MOF在提高基因编辑精度和效率方面的潜力.
  • 讨论基于MOF的系统作为先进的非病毒治疗输送载体.

主要方法:

  • 探索MOF设计策略,包括刺激响应链接器和生物活性连接物.
  • 对基于MOF的纳米平台进行分析,以有针对性地提供CRISPR/Cas9组件.
  • 对评估MOF性能与传统非病毒系统相比的比较研究的综述.
  • 讨论细胞内贩运和临床前治疗潜力.

主要成果:

  • MOFs表现出可调节的多孔性,高载荷能力和良好的生物相容性.
  • 基于MOF的系统可以在目标地点控制释放CRISPR/Cas9有效载荷.
  • 与传统载体相比,MOFs表现出优越的稳定性,转染效率和减少的目标外影响.
  • MOF输送系统显示出克服免疫性和细胞毒性问题的潜力.

结论:

  • 基于MOF的纳米平台代表了CRISPR/Cas9交付的变革性方法.
  • 这些系统为基因编辑疗法提供了更高的精度,效率和安全性.
  • MOFs为精准医学中先进的非病毒治疗输送系统铺平了道路.