Abstract:
Chimeric antigen receptor T cell (CAR-T) therapy has revolutionized the treatment approach for cancer, autoimmune disease, and heart disease. The integration of CAR into T cells is typically facilitated by retroviral or lentiviral vectors. However, the random insertion of CARs can lead to issues like clonal expansion, oncogenic transformation, variegated transgene expression, and transcriptional silencing. The advent of precise gene editing technology, like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), allows for controlled and precise genome modification, facilitating the translation of CAR-T research to the clinical applications. This review aims to provide a comprehensive analysis of the application of CRISPR gene editing techniques in the context of precise deletion and insertion methodologies, with a specific focus on their potential for enhancing the development and utilization of CAR-T cell therapy.
摘要:
嵌合抗原受体T细胞(CAR-T)疗法彻底改变了癌症的治疗方法,自身免疫性疾病,还有心脏病.通常通过逆转录病毒或慢病毒载体促进CAR整合到T细胞中。然而,CAR的随机插入会导致克隆扩增等问题,致癌转化,多样化的转基因表达,和转录沉默。精准基因编辑技术的出现,如簇状定期间隔短回文重复(CRISPR),允许控制和精确的基因组修饰,促进CAR-T研究转化为临床应用。这篇综述旨在全面分析CRISPR基因编辑技术在精确缺失和插入方法中的应用,特别关注它们在增强CAR-T细胞疗法开发和利用方面的潜力。