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利用 CRISPR/Cas9 技术构建抗 CD19 CAR 敲入人诱导多能干细胞系

英文原题:Generation of an anti-CD19 CAR knock-in human induced pluripotent stem cell line using CRISPR/Cas9 technology.

查看英文原题

Generation of an anti-CD19 CAR knock-in human induced pluripotent stem cell line using CRISPR/Cas9 technology.

PubMed 2025/04/26(内容时间) Stem Cell Res Q4 · IF 0.6(JCR 2025)

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中文摘要

CAR-T 细胞疗法是肿瘤免疫治疗领域的一项重大突破。靶向CD19的CAR-T 细胞已成为治疗B细胞恶性肿瘤的重要策略。本研究利用CRISPR/Cas9介导的基因靶向技术,成功构建了敲入抗CD19 CAR的人诱导多能干细胞(iPSC)系。该细胞系可稳定表达CAR基因,同时维持典型干细胞形态和正常核型。此外,该细胞系具有多谱系分化潜能,可定向分化为多种表达嵌合抗原受体、能够靶向CD19阳性肿瘤的免疫细胞。

展开英文摘要原文

Chimeric antigen receptor T cell (CAR-T) therapy represents a major breakthrough in the field of tumor immunotherapy. CD19-targeted CAR-T cells (CD19 CAR-T) have emerged as an important therapeutic approach for treating B-cell malignancies.

We successfully constructed a human induced pluripotent stem cell (iPSC) line with an anti-CD19 CAR knock-in using CRISPR/Cas9-mediated gene targeting technology. This cell line can stably express the CAR gene while maintaining its typical stem cell morphology and normal karyotype.

Furthermore, this cell line possesses multilineage differentiation potential and can be directionally differentiated into various chimeric antigen receptor-expressing immune cells for targeting CD19-positive tumors.

论文信息

作者
Wang S、Feng Y、Xing Q、Zhou T、Liu J
第一作者单位
Department of Hematology, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou 510630, China.China
通讯作者单位
Department of Hematology, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou 510630, China. Electronic address: liujiaj@mail.sysu.edu.cn.China
文献类型
非美国政府资助研究
期刊
Stem cell research2025 Aug
原文标识
PubMed 40311328 · DOI 10.1016/j.scr.2025.103721