CRISPR Cas系统：从细菌防御机制到重塑基因研究和翻译治疗的革命性工具

The Microbe Pub Date : 2025-04-24 DOI:10.1016/j.microb.2025.100344

Kamran Saeed , Faisal Ayub , Muhammad Arshad Durrani , Muhammad Mujahid

{"title":"CRISPR Cas系统：从细菌防御机制到重塑基因研究和翻译治疗的革命性工具","authors":"Kamran Saeed , Faisal Ayub , Muhammad Arshad Durrani , Muhammad Mujahid","doi":"10.1016/j.microb.2025.100344","DOIUrl":null,"url":null,"abstract":"<div><div>The CRISPR-Cas systems, initially identified as bacterial defense mechanisms, have become innovative tools for genetic research and translational therapeutics. These systems are made up of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins, which precisely target and cleave specific genome sequences, thus providing bacteria with immunity against viral infections. This review traces the evolution of CRISPR-Cas systems from their discovery as a bacterial defence mechanism to present uses in genetic engineering and beyond. The diversity of these systems, their structure and function, and the profound impact of these systems on genetic research, including their use in the creation of novel therapies for a variety of diseases, will be explored. In particular, this review discuss therapeutic applications of CRISPR in various neurodegenerative disorders (Alzheimer disease, Huntington’s disease and Parkinson’s disease), cardiovascular diseases, viral disorders including HIV, blood disorders (β-thalassemia, haemophilia and sickle cell anaemia), ocular disorders (Retinitis pigmentosa, Leber's congenital amaurosis, Aniridia, X-linked juvenile retinoschisis) mitochondrial disorders, Duchene muscular dystrophy and cancer along with the prospects concerning them. Furthermore, we discuss recent advancements, including novel Cas variants (CasΦ, CasX/Cas12f), precision editing technologies (base editing and prime editing), advanced delivery systems (nanoparticles, viral vectors), and emerging diagnostic platforms (SHERLOCK and DETECTR). Despite the extensive literature available on CRISPR-Cas systems, there remains a need for an updated review addressing recent developments, technological advancements, and future directions. This review aims to fill this gap by presenting an in-depth analysis of cutting-edge research, distinguishing itself from previous reviews by incorporating newly discovered CRISPR-Cas variants, emerging therapeutic strategies, and evolving regulatory landscapes.</div></div>","PeriodicalId":101246,"journal":{"name":"The Microbe","volume":"7 ","pages":"Article 100344"},"PeriodicalIF":0.0000,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"CRISPR Cas systems: From bacterial defense mechanisms to revolutionary tools reshaping genetic research and translation therapeutics\",\"authors\":\"Kamran Saeed , Faisal Ayub , Muhammad Arshad Durrani , Muhammad Mujahid\",\"doi\":\"10.1016/j.microb.2025.100344\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>The CRISPR-Cas systems, initially identified as bacterial defense mechanisms, have become innovative tools for genetic research and translational therapeutics. These systems are made up of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins, which precisely target and cleave specific genome sequences, thus providing bacteria with immunity against viral infections. This review traces the evolution of CRISPR-Cas systems from their discovery as a bacterial defence mechanism to present uses in genetic engineering and beyond. The diversity of these systems, their structure and function, and the profound impact of these systems on genetic research, including their use in the creation of novel therapies for a variety of diseases, will be explored. In particular, this review discuss therapeutic applications of CRISPR in various neurodegenerative disorders (Alzheimer disease, Huntington’s disease and Parkinson’s disease), cardiovascular diseases, viral disorders including HIV, blood disorders (β-thalassemia, haemophilia and sickle cell anaemia), ocular disorders (Retinitis pigmentosa, Leber's congenital amaurosis, Aniridia, X-linked juvenile retinoschisis) mitochondrial disorders, Duchene muscular dystrophy and cancer along with the prospects concerning them. Furthermore, we discuss recent advancements, including novel Cas variants (CasΦ, CasX/Cas12f), precision editing technologies (base editing and prime editing), advanced delivery systems (nanoparticles, viral vectors), and emerging diagnostic platforms (SHERLOCK and DETECTR). Despite the extensive literature available on CRISPR-Cas systems, there remains a need for an updated review addressing recent developments, technological advancements, and future directions. This review aims to fill this gap by presenting an in-depth analysis of cutting-edge research, distinguishing itself from previous reviews by incorporating newly discovered CRISPR-Cas variants, emerging therapeutic strategies, and evolving regulatory landscapes.</div></div>\",\"PeriodicalId\":101246,\"journal\":{\"name\":\"The Microbe\",\"volume\":\"7 \",\"pages\":\"Article 100344\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-04-24\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"The Microbe\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2950194625001128\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"The Microbe","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2950194625001128","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}

引用次数: 0

摘要

CRISPR-Cas系统最初被确定为细菌防御机制，现已成为基因研究和转化治疗的创新工具。这些系统由聚集规律间隔的短回文重复（CRISPR）和CRISPR相关（Cas）蛋白组成，它们精确地靶向和切割特定的基因组序列，从而为细菌提供抵抗病毒感染的免疫力。这篇综述追溯了CRISPR-Cas系统的进化，从它们被发现作为一种细菌防御机制，到目前在基因工程和其他领域的应用。这些系统的多样性，它们的结构和功能，以及这些系统对遗传研究的深远影响，包括它们在创造各种疾病的新疗法中的应用，将被探索。本综述特别讨论了CRISPR在各种神经退行性疾病（阿尔茨海默病、亨廷顿病和帕金森病）、心血管疾病、病毒性疾病（包括HIV）、血液疾病（β-地中海贫血、血友病和镰状细胞贫血）、眼部疾病（色素性视网膜炎、利伯氏先天性黑内障、无虹膜、x连锁青少年视网膜裂）、线粒体疾病、杜氏肌营养不良症和癌症及其前景。此外，我们还讨论了最近的进展，包括新的Cas变体（CasΦ， CasX/Cas12f），精确编辑技术（碱基编辑和主编辑），先进的传递系统（纳米颗粒，病毒载体）和新兴的诊断平台（SHERLOCK和DETECTR）。尽管有大量关于CRISPR-Cas系统的文献，但仍然需要针对最近的发展、技术进步和未来方向进行更新的综述。本综述旨在通过对前沿研究的深入分析来填补这一空白，通过结合新发现的CRISPR-Cas变体、新兴的治疗策略和不断发展的监管格局，将其与以往的综述区分开来。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

CRISPR Cas systems: From bacterial defense mechanisms to revolutionary tools reshaping genetic research and translation therapeutics

The CRISPR-Cas systems, initially identified as bacterial defense mechanisms, have become innovative tools for genetic research and translational therapeutics. These systems are made up of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins, which precisely target and cleave specific genome sequences, thus providing bacteria with immunity against viral infections. This review traces the evolution of CRISPR-Cas systems from their discovery as a bacterial defence mechanism to present uses in genetic engineering and beyond. The diversity of these systems, their structure and function, and the profound impact of these systems on genetic research, including their use in the creation of novel therapies for a variety of diseases, will be explored. In particular, this review discuss therapeutic applications of CRISPR in various neurodegenerative disorders (Alzheimer disease, Huntington’s disease and Parkinson’s disease), cardiovascular diseases, viral disorders including HIV, blood disorders (β-thalassemia, haemophilia and sickle cell anaemia), ocular disorders (Retinitis pigmentosa, Leber's congenital amaurosis, Aniridia, X-linked juvenile retinoschisis) mitochondrial disorders, Duchene muscular dystrophy and cancer along with the prospects concerning them. Furthermore, we discuss recent advancements, including novel Cas variants (CasΦ, CasX/Cas12f), precision editing technologies (base editing and prime editing), advanced delivery systems (nanoparticles, viral vectors), and emerging diagnostic platforms (SHERLOCK and DETECTR). Despite the extensive literature available on CRISPR-Cas systems, there remains a need for an updated review addressing recent developments, technological advancements, and future directions. This review aims to fill this gap by presenting an in-depth analysis of cutting-edge research, distinguishing itself from previous reviews by incorporating newly discovered CRISPR-Cas variants, emerging therapeutic strategies, and evolving regulatory landscapes.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

The Microbe

自引率

0.00%

发文量