相关实验视频

Updated: Jan 17, 2026

Production of Human CRISPR-Engineered CAR-T Cells

Production of Human CRISPR-Engineered CAR-T Cells

Published on: March 15, 2021

通过CRISPR增强的CAR T细胞免疫疗法的系统发现

Paul Datlinger^1,2, Eugenia V Pankevich³, Cosmas D Arnold³

¹CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria. paul.datlinger@cellfie.org.

|September 24, 2025

概括

研究人员开发了CRISPR查平台CELLFIE, 显著提高抗白血病的CAR T细胞疗效,改善癌症免疫疗法.

科学领域:

免疫学
遗传学
生物技术

背景情况:

对于血液癌症来说,CAR T细胞疗法是有前途的,但由于T细胞功能障碍而面临挑战.
提高CAR T细胞功能对于改善治疗结果和克服耐药性至关重要.

研究的目的:

开发一个CRISPR查平台 (CELLFIE),用于识别增强CAR T细胞的基因修饰.
发现改善CAR T细胞增殖,持久性和抗白血病活性的新基因标.

主要方法:

对具有各种功能读数的人类初级CAR T细胞进行了全基因组CRISPR选.
在人体白血病异种移植模型中使用CROP- seq以优先选择查结果.
包括RHOG和FAS在内的确定的基因淘汰的广泛验证在各种模型和患者衍生细胞中进行.

主要成果:

在CELLFIE平台上成功发现了增强CAR T细胞功能,包括增殖和目标识别的基因标.
单独使用和与FAS结合使用,RHOG敲除已成为CAR T细胞功效的强效增强剂.
在临床前模型中,与标准的CAR T细胞相比,验证的CRISPR提升的CAR T细胞表现出更高的性能.

结论:

通过基因工程优化CAR-T细胞疗法.
RHOG绝杀是一种增强血液恶性瘤的CAR T细胞免疫疗法的有希望的策略.
这项研究为开发下一代高效的基于细胞的免疫疗法提供了基础资源.

更多相关视频

Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells

Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells

Published on: July 22, 2019

Performing an In Vitro Genome-Wide CRISPR Knockout Screen in Chimeric Antigen Receptor T Cells

Performing an In Vitro Genome-Wide CRISPR Knockout Screen in Chimeric Antigen Receptor T Cells

Published on: January 31, 2025

相关概念视频

Tumor Immunotherapy

Tumor Immunotherapy

Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.

CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...