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CRISPR/Cas 介导的小鼠基因组编辑用于开发药物递送机制。

CRISPR/Cas-mediated genome editing in mice for the development of drug delivery mechanism.

机构信息

Department of Biotechnology and Genetics, M.S. Ramaiah College of Arts, Science and Commerce, 7th Main Rd, MSRIT, M S R Nagar, Mathikere, Bengaluru, Karnataka, 560 054, India.

Department of Biochemistry, M.S. Ramaiah College of Arts, Science and Commerce, 7th Main Rd, MSRIT, M S R Nagar, Mathikere, Bengaluru, Karnataka, 560 054, India.

出版信息

Mol Biol Rep. 2023 Sep;50(9):7729-7743. doi: 10.1007/s11033-023-08659-z. Epub 2023 Jul 12.

Abstract

BACKGROUND

To manipulate particular locations in the bacterial genome, researchers have recently resorted to a group of unique sequences in bacterial genomes that are responsible for safeguarding bacteria against bacteriophages. Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (Cas9) are two such systems, each of which consists of an RNA component and an enzyme component.

METHODS AND RESULTS

This review focuses primarily on how CRISPR/Cas9 technology can be used to make models to study human diseases in mice. Creating RNA molecules that direct endonucleases to a specific position in the genome are crucial for achieving a specific genetic modification. CRISPR/Cas9 technology has allowed scientists to edit the genome with greater precision than ever before. Researchers can use knock-in and knock-out methods to model human diseases such as Neurological, cardiovascular disease, and cancer.

CONCLUSIONS

In terms of developing innovative methods to discover ailments for diseases/disorders, improved CRISPR/Cas9 technology will provide easier access to valuable novel animal models.

摘要

背景

为了操纵细菌基因组中的特定位置,研究人员最近求助于细菌基因组中一组负责保护细菌免受噬菌体侵害的独特序列。成簇规律间隔短回文重复序列 (CRISPR) 和 CRISPR 相关蛋白 9 (Cas9) 是两种这样的系统,每个系统都由 RNA 成分和酶成分组成。

方法和结果

本综述主要关注 CRISPR/Cas9 技术如何可用于在小鼠中建立模型来研究人类疾病。创建引导内切酶到基因组中特定位置的 RNA 分子对于实现特定的遗传修饰至关重要。CRISPR/Cas9 技术使科学家能够以前所未有的精度编辑基因组。研究人员可以使用敲入和敲除方法来模拟 Neurological、心血管疾病和癌症等人类疾病。

结论

在开发用于发现疾病/病症的创新方法方面,改进的 CRISPR/Cas9 技术将为获得有价值的新型动物模型提供更便捷的途径。

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