基于CRISPR的基因组编辑器的药物输送系统
Victoria Madigan1,2,3,4,5, Feng Zhang1,2,3,4,5, James E Dahlman6
1Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Nature reviews. Drug discovery
|September 18, 2023
概括
修改基因的CRISPR药物需要有效的输送系统才能安全地进入细胞. 本综述涵盖了腺相关病毒和脂质纳米粒子,检查了它们在CRISPR传递方面的工程,临床前和临床应用.
科学领域:
- 生物技术是生物技术.
- 遗传学 是一个遗传学.
- 药物运输 药物运输 药物运输
背景情况:
- 基于CRISPR的疗法提供了精确的基因操纵.
- 有效和安全地将CRISPR系统输入目标细胞仍然是一个重大挑战.
- 免疫反应和非目标输送需要复杂的输送工具.
研究的目的:
- 对基于CRISPR的基因组编辑器的当前药物递送系统进行审查.
- 专注于腺相关病毒和脂质纳米粒子.
- 讨论未来的临床前向CRISPR交付策略.
主要方法:
- 工程化腺相关病毒和脂质纳米颗粒的审查.
- 对临床前动物模型表征数据的分析.
- 突出了近期对CRISPR传递系统的临床试验数据.
- 讨论使用聚合物,蛋白质和病毒样颗粒的临床前向.
主要成果:
- 基相关病毒和脂质纳米颗粒是CRISPR技术的主要输送系统.
- 在临床前模型中的工程和表征为临床翻译提供了信息.
- 早期临床试验数据为这些系统的安全性和有效性提供了洞察力.
- 多种临床前策略对未来的CRISPR交付有希望.
结论:
- 传递系统对于基于CRISPR的疗法的临床成功至关重要.
- 基相关病毒和脂质纳米颗粒已经建立但正在发展的传递平台.
- 正在进行的对新型输送车辆的研究将扩大对遗传疾病的治疗选择.
更多相关视频
相关概念视频
CRISPR/Cas9 Genome Editing
48
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...
48
CRISPR
52.2K
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...
52.2K
CRISPR and crRNAs
17.1K
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...
17.1K


