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相关概念视频

CRISPR01:59

CRISPR

50.6K
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...
50.6K
CRISPR and crRNAs02:53

CRISPR and crRNAs

17.0K
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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相关实验视频

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Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles "Nanoblades"
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功能化的细胞外纳米纤维作为先进的CRISPR输送系统.

Siqing Wang1, Huimin Kong1, Chenya Zhuo1

  • 1Laboratory of Biomaterials and Translational Medicine, Center for Nanomedicine, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou 510630, China. zhuochy5@mail.sysu.edu.cn.

Biomaterials science
|May 29, 2024
PubMed
概括

细胞外纳米纤维 (EVs) 为提供CRISPR基因编辑工具提供了一个有希望的解决方案,克服了遗传疾病治疗目前的交付挑战. 研究探讨了各种功能化的EV和工程技术,以改善临床翻译.

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Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection
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科学领域:

  • 生物技术是生物技术.
  • 纳米医学是一种纳米医学.
  • 基因治疗 基因治疗

背景情况:

  • 集群定期间隔的短时间Palindromic重复 (CRISPR) 系统显示出治疗遗传疾病的巨大潜力.
  • 对CRISPR技术的有效交付策略至关重要,但目前对于临床应用来说发展不足.

研究的目的:

  • 为了总结功能性的细胞外纳米纤维 (EVs) 的CRISPR交付.
  • 为了检查各种EV类型的细胞内通路.
  • 概述EV功能化的技术,并讨论其临床转化.

主要方法:

  • 对用于CRISPR传递的不同类型的功能性EV进行审查 (未修改,修改,工程病毒样颗粒,外体-脂质体混合体).
  • 检查与这些EVs相关的明显细胞内通路.
  • 用于EV功能化的尖端技术概述 (生产细胞,囊泡和病毒样粒子工程).

主要成果:

  • 功能性电动汽车具有诸如高生物相容性,生物透性和低免疫性等优点.
  • 各种EV工程技术使CRISPR交付能力多样化.
  • 确定了功能化电动车的临床转化所面临的挑战和拟议的设计策略.

结论:

  • 功能化的细胞外纳米纤维是CRISPR交付的一个有希望的平台.
  • 需要进一步的开发和合理的设计,以使基于EV的CRISPR疗法的临床转化成功.