{"title":"CRISPR-Cas技术及其应用的进展：革新精准医学。","authors":"Sarkar Sardar Azeez, Rahin Shareef Hamad, Bahra Kakamin Hamad, Mudhir Sabir Shekha, Peter Bergsten","doi":"10.3389/fgeed.2024.1509924","DOIUrl":null,"url":null,"abstract":"<p><p>CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins) has undergone marked advancements since its discovery as an adaptive immune system in bacteria and archaea, emerged as a potent gene-editing tool after the successful engineering of its synthetic guide RNA (sgRNA) toward the targeting of specific DNA sequences with high accuracy. Besides its DNA editing ability, further-developed Cas variants can also edit the epigenome, rendering the CRISPR-Cas system a versatile tool for genome and epigenome manipulation and a pioneering force in precision medicine. This review explores the latest advancements in CRISPR-Cas technology and its therapeutic and biomedical applications, highlighting its transformative impact on precision medicine. Moreover, the current status of CRISPR therapeutics in clinical trials is discussed. Finally, we address the persisting challenges and prospects of CRISPR-Cas technology.</p>","PeriodicalId":73086,"journal":{"name":"Frontiers in genome editing","volume":"6 ","pages":"1509924"},"PeriodicalIF":4.9000,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11669675/pdf/","citationCount":"0","resultStr":"{\"title\":\"Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine.\",\"authors\":\"Sarkar Sardar Azeez, Rahin Shareef Hamad, Bahra Kakamin Hamad, Mudhir Sabir Shekha, Peter Bergsten\",\"doi\":\"10.3389/fgeed.2024.1509924\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins) has undergone marked advancements since its discovery as an adaptive immune system in bacteria and archaea, emerged as a potent gene-editing tool after the successful engineering of its synthetic guide RNA (sgRNA) toward the targeting of specific DNA sequences with high accuracy. Besides its DNA editing ability, further-developed Cas variants can also edit the epigenome, rendering the CRISPR-Cas system a versatile tool for genome and epigenome manipulation and a pioneering force in precision medicine. This review explores the latest advancements in CRISPR-Cas technology and its therapeutic and biomedical applications, highlighting its transformative impact on precision medicine. Moreover, the current status of CRISPR therapeutics in clinical trials is discussed. Finally, we address the persisting challenges and prospects of CRISPR-Cas technology.</p>\",\"PeriodicalId\":73086,\"journal\":{\"name\":\"Frontiers in genome editing\",\"volume\":\"6 \",\"pages\":\"1509924\"},\"PeriodicalIF\":4.9000,\"publicationDate\":\"2024-12-12\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11669675/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Frontiers in genome editing\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.3389/fgeed.2024.1509924\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q1\",\"JCRName\":\"BIOTECHNOLOGY & APPLIED MICROBIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Frontiers in genome editing","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.3389/fgeed.2024.1509924","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
CRISPR-Cas （Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins）自被发现作为细菌和古细菌的适应性免疫系统以来，已经取得了显著的进展，在其合成的引导RNA （sgRNA）成功地用于高精度靶向特定DNA序列后，它成为了一种强有力的基因编辑工具。除了具有DNA编辑能力外，进一步开发的Cas变体还可以编辑表观基因组，使CRISPR-Cas系统成为基因组和表观基因组操作的多功能工具，并成为精准医学的先驱力量。本文综述了CRISPR-Cas技术及其治疗和生物医学应用的最新进展，重点介绍了其对精准医学的变革性影响。此外，还讨论了CRISPR治疗方法在临床试验中的现状。最后，我们讨论了CRISPR-Cas技术持续存在的挑战和前景。
Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine.
CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins) has undergone marked advancements since its discovery as an adaptive immune system in bacteria and archaea, emerged as a potent gene-editing tool after the successful engineering of its synthetic guide RNA (sgRNA) toward the targeting of specific DNA sequences with high accuracy. Besides its DNA editing ability, further-developed Cas variants can also edit the epigenome, rendering the CRISPR-Cas system a versatile tool for genome and epigenome manipulation and a pioneering force in precision medicine. This review explores the latest advancements in CRISPR-Cas technology and its therapeutic and biomedical applications, highlighting its transformative impact on precision medicine. Moreover, the current status of CRISPR therapeutics in clinical trials is discussed. Finally, we address the persisting challenges and prospects of CRISPR-Cas technology.
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