CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:CRISPR-Cas and CRISPR-based screening system for precise gene editing and targeted cancer therapy.
CRISPR-Cas and CRISPR-based screening system for precise gene editing and targeted cancer therapy.
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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。
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.
在 PubMed 查看 → 出版商原文(DOI) 全文 PDF(PMC)· 可下载 治疗专题与资料阅读指南 资料来源与翻译说明 报告译文或资料问题 →
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