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.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Genome Editing for Engineering the Next Generation of Advanced Immune Cell Therapies.
Genome Editing for Engineering the Next Generation of Advanced Immune Cell Therapies.
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我们目前通过合成生物学和基因组编辑所具备的基因工程能力,是生物医学科学一场革命的基础:利用基因编程的细胞作为治疗手段。这一范式最典型的例子是过继转移经基因工程改造以表达肿瘤特异性受体的T细胞,例如CAR或工程化TCR。该方法已在原本无法治愈的血液系统恶性肿瘤患者中带来了前所未有的完全缓解率。然而,该方法对实体瘤——占绝大多数肿瘤——仍基本无效。此外,该疗法的自体性质以及癌细胞与T细胞之间共享标志物所带来的局限性,进一步限制了这些疗法的可及性。在此,我们描述了如何应用前沿基因组编辑方法来释放这些革命性疗法的全部潜力,从而提高治疗效果和患者可及性。
Our current genetic engineering capacity through synthetic biology and genome editing is the foundation of a revolution in biomedical science: the use of genetically programmed cells as therapeutics. The prime example of this paradigm is the adoptive transfer of genetically engineered T cells to express tumor-specific receptors, such as chimeric antigen receptors (CARs) or engineered T-cell receptors (TCR). This approach has led to unprecedented complete remission rates in patients with otherwise incurable hematological malignancies.
However, this approach is still largely ineffective against solid tumors, which comprise the vast majority of neoplasms. Also, limitations associated with the autologous nature of this therapy and shared markers between cancer cells and T cells further restrict the access to these therapies.
Here, we described how cutting-edge genome editing approaches have been applied to unlock the full potential of these revolutionary therapies, thereby increasing therapeutic efficacy and patient accessibility.
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