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用于精准基因编辑与靶向肿瘤治疗的 CRISPR-Cas 及基于 CRISPR 的筛选系统

英文原题:CRISPR-Cas and CRISPR-based screening system for precise gene editing and targeted cancer therapy.

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CRISPR-Cas and CRISPR-based screening system for precise gene editing and targeted cancer therapy.

PubMed 2024/05/30(内容时间) J Transl Med Q1 · IF 9.7(JCR 2025)

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中文摘要

基于 CRISPR(成簇规律间隔短回文重复序列)-Cas 系统的发现,靶向癌症治疗已用于临床癌症治疗。这项前沿技术将癌症研究推进至分子层面,目前广泛应用于遗传学研究和临床精准癌症治疗。本综述总结 CRISPR/Cas 和 CRISPR 筛选系统用于基因改造的研究,讨论成功开展 CRISPR 筛选的关键组成部分,包括 Cas 酶、向导 RNA(gRNA)文库及靶细胞或器官。此外,我们重点介绍 CRISPR 筛选系统在体外和体内 CAR-T 细胞疗法、药物靶点发现、药物筛选及药物选择中的应用。综述还阐述 CRISPR 系统在精准临床医学中的优势和潜在障碍,并展望未来基因疗法。总之,本文从全面且实用的角度探讨 CRISPR/Cas 及 CRISPR 筛选系统在治疗癌症缺陷方面的发展,旨在进一步提高临床治疗及个体化基因治疗的精准性。

展开英文摘要原文

Target cancer therapy has been developed for clinical cancer treatment based on the discovery of CRISPR (clustered regularly interspaced short palindromic repeat) -Cas system. This forefront and cutting-edge scientific technique improves the cancer research into molecular level and is currently widely utilized in genetic investigation and clinical precision cancer therapy.

In this review, we summarized the genetic modification by CRISPR/Cas and CRISPR screening system, discussed key components for successful CRISPR screening, including Cas enzymes, guide RNA (gRNA) libraries, target cells or organs.

Furthermore, we focused on the application for CAR-T cell therapy, drug target, drug screening, or drug selection in both ex vivo and in vivo with CRISPR screening system.

In addition, we elucidated the advantages and potential obstacles of CRISPR system in precision clinical medicine and described the prospects for future genetic therapy. In summary, we provide a comprehensive and practical perspective on the development of CRISPR/Cas and CRISPR screening system for the treatment of cancer defects, aiming to further improve the precision and accuracy for clinical treatment and individualized gene therapy.

论文信息

作者
Qin M、Deng C、Wen L、Luo G、Meng Y
第一作者单位
Reproductive Medical Center, Affiliated Foshan Maternity & Child Healthcare Hospital, Southern Medical University (Foshan Women and Children Hospital), Foshan, Guangdong, 528000, China.China
通讯作者单位
Guangdong Provincial Key Laboratory of Tumor Interventional Diagnosis and Treatment, Zhuhai People's Hospital, Zhuhai Clinical Medical College of Jinan University, Zhuhai, Guangdong, 519000, China. mengya_85@163.com.China
文献类型
综述 · 非美国政府资助研究
期刊
Journal of translational medicine2024 May 30
原文标识
PubMed 38816739 · DOI 10.1186/s12967-024-05235-2