基于分支DNA的自组平台用于sgRNA/Cas9/Antisense传输
Jianbing Liu1, Tiantian Wu1,2, Xuehe Lu1,3
1CAS Key Laboratory of Nanosystem and Hierarchical Fabrication, CAS Center for Excellence in Nanoscience , National Center for Nanoscience and Technology , Beijing 100190 , China.
Journal of the American Chemical Society
|November 16, 2019
概括
这项研究介绍了一种新的分支DNA纳米平台,用于编码基因编辑和基因沉默疗法. 这种生物相容的纳米载体有效向瘤,
科学领域:
- 生物技术
- 纳米医学
- 分子疗法
背景情况:
- DNA纳米结构提供了针对性治疗的潜力.
- 开发用于组合基因编辑和沉默的多功能平台对于协同治疗癌症至关重要.
研究的目的:
- 开发一种用于构建基因编辑 (sgRNA/Cas9) 和基因沉默 (反意义) 剂的分支DNA纳米平台的简单策略.
- 评估这种纳米平台在以PLK1为向的协同瘤治疗中的 in vitro 和 in vivo 疗效.
主要方法:
- 通过自组装构建分支DNA纳米结构.
- 针对PLK1的sgRNA/Cas9/antisense复合物的加载.
- 使用宿主-客人相互作用将一个活跃的向性和一个内体逃生结合起来.
主要成果:
- DNA纳米平台有效地装载了治疗复合体.
- 向阿普坦和内体逃生的结合增强了输送.
- 在体外和体内显著抑制瘤生长.
- 没有明显的全身毒性.
结论:
- 开发的分支DNA纳米平台能够有效地提供基因编辑和沉默剂.
- 这种多功能纳米载体显示出协同瘤治疗的前景.
- 提供了开发先进基因疗法的新策略.
相关概念视频
CRISPR/Cas9 Genome Editing
1.5K
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...
1.5K
CRISPR
57.3K
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...
57.3K
CRISPR and crRNAs
18.6K
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...
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...
18.6K


