一种温度敏感和干扰素无声的仙台病毒载体,用于CRISPR-Cas9传递和基因编辑在人类初级细胞中
Christian S Stevens1, Jillian Carmichael1, Ruth Watkinson1
1Department of Microbiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029.
bioRxiv : the preprint server for biology
|May 15, 2024
概括
一种对温度敏感的新型仙台病毒 (ts SeV) 载体有效地提供了CRISPR-Cas9基因编辑工具. 这种安全的方法在干细胞中实现了高的编辑率,抑制了HIV-1感染.
科学领域:
- 基因治疗 基因治疗
- 病毒学 病毒学
- 免疫学 免疫学 免疫学
背景情况:
- 基因编辑技术需要安全有效的传递系统用于治疗应用.
- 目前的输送方法可以诱导免疫反应,限制它们在敏感细胞类型中的使用.
结论:
- ts SeV平台提供了一种安全,高效和灵活的基因编辑工具交付方法.
- 这种方法显示了个性化医疗和治疗遗传疾病的潜力.
- tsSeV最大限度地减少干扰素反应,使敏感细胞类型的基因编辑成为可能.
关键词:
CCR5 CCR5 的意思是什么?这就是CRISPR/Cas9的作用.基因编辑 基因编辑艾滋病病毒 艾滋病病毒 艾滋病病毒这就是Paramyxoviridae.仙台病毒的病毒造血干细胞和原始细胞.病毒载体病毒载体更多相关视频
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相关概念视频
01:59CRISPR
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 Short...
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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...