人类神经器官和组合体中的CRISPR屏幕
Xiangling Meng1,2, Noah Reis1,2, Michael C Bassik3
1Department of Psychiatry and Behavioral Sciences, Stanford University, Stanford, CA, USA.
Nature protocols
|December 19, 2025
概括
这项研究引入了一种新的协议,将CRISPR查与神经器官结合起来,以映射影响人类神经发育的疾病基因. 这种方法加速了对大脑发育和疾病机制的理解.
科学领域:
- 神经科学是一个神经科学.
- 发展生物学 发展生物学
- 遗传学 遗传学 是一个
背景情况:
- 了解人类的神经发育和相关疾病是复杂的.
- 多能干细胞和基因组编辑提供了新的研究途径.
- 器官体和组合体等体外模型允许研究人类神经发育.
研究的目的:
- 提出一个详细的协议,将合并的CRISPR-Cas9查与神经器官和组合体模型集成在一起.
- 展示该协议在将疾病基因映射到细胞通路和神经发育过程中的应用.
- 促进对人类大脑发育和疾病机制的研究.
主要方法:
- 结合合集的CRISPR-Cas9查与人类神经器官和集合体模型.
- 基因干扰,查和候选基因验证的详细协议.
- 利用干细胞培养,神经分化,基因工程,FACS和下一代测序方面的专业知识.
主要成果:
- 该协议可以将数百种疾病基因映射到特定的细胞通路上.
- 它允许研究神经元的生成,迁移和整合在人类大脑发育模型.
- 查实验提供了对基因在神经发育和疾病中的作用的见解.
结论:
- 基因查和人类细胞模型的综合平台对研究大脑发育和疾病具有强大作用.
- 这种方法加快了疾病机制和潜在治疗点的发现.
- 它为神经系统疾病的新疗法发现铺平了道路.
相关概念视频
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 Short...
Homologous Recombination
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...
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...
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...
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 Short...
The Antiviral System of Bacteria and Archaea: CRISPR
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats is a adaptive immune system found in bacteria and archaea that protects against viral infections. This system enables prokaryotic cells to identify, remember, and neutralize foreign genetic elements, primarily bacteriophages, by storing fragments of the invader’s DNA as a genetic memory.The CRISPR immune response begins during an initial infection. Cas (CRISPR-associated) proteins play a central role in this defense.
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...


