相关实验视频

Updated: Jul 13, 2025

Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection

Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection

Published on: May 23, 2016

使用工程外细胞囊泡进行CRISPR-Cas9传递策略

Yaoyao Lu¹, Kelly Godbout¹, Gabriel Lamothe¹

¹Centre de Recherche du CHU de Québec -Université Laval, Québec city, QC G1V4G2, Canada.

Molecular therapy. Nucleic acids

|October 16, 2023

概括

细胞外囊泡 (EVs) 显示出提供CRISPR-Cas9基因编辑工具的希望. 本综述探讨了增强EV负载和向有效遗传疾病治疗的方法.

科学领域:

生物技术是生物技术.
分子生物学分子生物学
遗传学遗传学是一个

背景情况:

治疗性基因组编辑通过纠正突变,为遗传疾病提供潜在的治疗方法.
由于其效率和多功能性,CRISPR-Cas9系统是先进的基因编辑工具.
将CRISPR-Cas9安全有效地输送到目标器官仍然是基因疗法的重大挑战.

研究的目的:

审查最近利用细胞外囊泡 (EVs) 进行CRISPR-Cas9传递的进展.
突出提高电动汽车装载,表面功能化和准效率的策略.
讨论EV介导的CRISPR-Cas9在治疗遗传疾病方面的潜力.

主要方法:

关于细胞外囊泡 (EV) 生物发生和功能的当前文献的综述.
对将CRISPR-Cas9组件装入电动汽车的方法进行分析.
检查表面修改技术以改善EV准.
评估提高CRISPR-Cas9向特定组织和细胞传递效率的策略.

主要成果:

细胞外囊泡 (EVs) 可以自然地运输治疗分子,包括CRISPR-Cas9系统.
目前正在开发各种加载技术,以在电动汽车中封装CRISPR-Cas9.
电动体的表面功能化可以提高其特异性和目标细胞的吸收.
优化的EV传递系统显示出高效的 in vivo 基因编辑的潜力.

结论:

细胞外囊泡 (EVs) 是一个有前途的平台,可以针对CRISPR-Cas9基因编辑技术的目标提供.
电动汽车装载和功能化的进步对于克服当前的交付障碍至关重要.
通过EV介导的CRISPR-Cas9传递对遗传医学和疾病治疗的未来有很大的潜力.

关键词:

克里斯普尔-Cas9是什么意思 MT:利用细胞外囊泡作为治疗剂特别版送货送货送货送货送货送货细胞外囊泡中的细胞外囊泡.基因治疗的基因疗法组织向向组织向.

更多相关视频

Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles "Nanoblades"

Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles "Nanoblades"

Published on: March 31, 2021

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Published on: March 29, 2019

相关概念视频

CRISPR

CRISPR

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...

CRISPR/Cas9 Genome Editing

CRISPR/Cas9 Genome Editing

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...

CRISPR and crRNAs

CRISPR and crRNAs

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...