相关实验视频
Updated: Jun 9, 2025

07:56
Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
21.5K
对抗CRISPR蛋白的计算指导高通量工程用于人类细胞中精确的基因组编辑
Julia Marsiglia1, Kia Vaalavirta1, Estefany Knight1
1Acrobat Genomics, Inc., San Carlos, CA 94070, USA.
Cell reports methods
|October 22, 2024
概括
科学家们设计了抗CRISPR蛋白质 (Acrs),以提高CRISPR基因编辑的精度. 这种方法通过减少非目标编辑来提高基因编辑疗法的安全性.
科学领域:
- 遗传学 是一个遗传学.
- 分子生物学分子生物学
- 生物技术是生物技术.
背景情况:
- 克里斯普尔-卡斯系统是具有治疗潜力的强大基因组编辑工具.
- 非目标突变是限制CRISPR临床应用的一个重大挑战.
- 菌体利用抗CRISPR蛋白 (Acrs) 来抑制CRISPR-Cas的活性.
研究的目的:
- 设计抗CRISPR蛋白质 (Acrs),以提高CRISPR-Cas基因组编辑的精度.
- 开发一个可扩展的选框架,以提高工程 Acrs 的特异性.
主要方法:
- 利用计算指导,深度突变扫描和高通量DNA修复测量.
- 在一个单一的实验中测试了大约1万种反CRISPR变异.
- 在人类细胞中验证了有希望的变体,并评估了与高可靠性Cas9.9的协同作用.
主要成果:
- 确定了Acr变种,显著提高了基因编辑精度.
- 证明了对编辑特异性的强有力的增强.
- 在工程 Acrs 和高保真 Cas9 变体之间观察到协同效应.
结论:
- 开发了一个可扩展的查框架,用于工程 Acrs 以提高基因编辑精度.
- 设计的AcrIIA4变种增强了CRISPR-Cas编辑特异性.
- 这种方法有望提高基因编辑疗法的安全性.
相关概念视频
CRISPR
49.7K
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...
49.7K
Homologous Recombination
50.2K
The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
50.2K
CRISPR and crRNAs
16.8K
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...
16.8K

