Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington 98109, USA.
Department of Bioengineering and Molecular Engineering & Sciences Institute, University of Washington, Seattle, Washington 98195, USA; email:
Annu Rev Biomed Eng. 2021 Jul 13;23:385-405. doi: 10.1146/annurev-bioeng-070620-033348. Epub 2021 Apr 16.
Gene therapy makes it possible to engineer chimeric antigen receptors (CARs) to create T cells that target specific diseases. However, current approaches require elaborate and expensive protocols to manufacture engineered T cells ex vivo, putting this therapy beyond the reach of many patients who might benefit. A solution could be to program T cells in vivo. Here, we evaluate the clinical need for in situ CAR T cell programming, compare competing technologies, review current progress, and provide a perspective on the long-term impact of this emerging and rapidly flourishing biotechnology field.
基因治疗使得设计嵌合抗原受体(CAR)来制造靶向特定疾病的 T 细胞成为可能。然而,目前的方法需要精心设计和昂贵的方案来体外制造工程化的 T 细胞,这使得许多可能受益的患者无法获得这种治疗。一个解决方案可能是在体内编程 T 细胞。在这里,我们评估了体内 CAR T 细胞编程的临床需求,比较了竞争技术,回顾了当前的进展,并对这个新兴且迅速发展的生物技术领域的长期影响提供了一个视角。