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GMP 生产的 CRISPR/Cas9 技术作为支持肿瘤免疫治疗的优势工具

英文原题:GMP-manufactured CRISPR/Cas9 technology as an advantageous tool to support cancer immunotherapy.

PubMed 2024/03/01(内容时间) J Exp Clin Cancer Res Q1 · IF 14.3(JCR 2025)

研究概要

在此,我们将全面汇集用于支持癌症治疗的实际GMP级CRISPR/Cas9介导方法,重点展示该技术如何为治疗肿瘤开辟新机遇。

研究思路结论见上方概要

用于治疗人类相关疾病的 CRISPR/Cas9 系统已取得显著成果,尽管其在癌症研究中的潜在应用正在不断提升,但该方法在临床实践中的应用仍是一项新兴技术。 主体部分:CRISPR/Cas9 技术尚未作为单一疗法用于治疗肿瘤,但可与传统治疗策略相结合,为患者提供个性化基因治疗。与化疗、放疗和免疫治疗的联合已被证明是筛选、识别、验证和纠正肿瘤靶点的有力手段。近年来,CRISPR/Cas9 技术与 CAR T-cell 疗法已被整合,为所有患者生产更高效的 CAR T-cells 开辟了新机遇。目前,若干临床级系统已有符合 GMP 的设备和试剂可用,为加速开发新型治疗方法奠定了基础和框架。

展开英文摘要原文

BACKGROUND: CRISPR/Cas9 system to treat human-related diseases has achieved significant results and, even if its potential application in cancer research is improving, the application of this approach in clinical practice is still a nascent technology. MAIN BODY: CRISPR/Cas9 technology is not yet used as a single therapy to treat tumors but it can be combined with traditional treatment strategies to provide personalized gene therapy for patients. The combination with chemotherapy, radiation and immunotherapy has been proven to be a powerful means of screening, identifying, validating and correcting tumor targets. Recently, CRISPR/Cas9 technology and CAR T-cell therapies have been integrated to open novel opportunities for the production of more efficient CAR T-cells for all patients. GMP-compatible equipment and reagents are already available for several clinical-grade systems at present, creating the basis and framework for the accelerated development of novel treatment methods. CONCLUSION: Here we will provide a comprehensive collection of the actual GMP-grade CRISPR/Cas9-mediated approaches used to support cancer therapy highlighting how this technology is opening new opportunities for treating tumors.

论文信息

作者
Caforio M、Iacovelli S、Quintarelli C、Locatelli F、Folgiero V
第一作者单位
U.O. Cellular and Genetic Therapy of Hematological Diseases, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.Italy
通讯作者单位
U.O. Cellular and Genetic Therapy of Hematological Diseases, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. valentina.folgiero@opbg.net.Italy
文献类型
综述
期刊
Journal of experimental & clinical cancer research : CR2024 Mar 1
原文标识
PubMed 38424590 · DOI 10.1186/s13046-024-02993-1