Yong Seok-Beom, Chung Jee Young, Song Yoonsung, Kim Yong-Hee
1Department of Bioengineering, Hanyang University, Seoul, 04763 Republic of Korea.
3BK 21 Plus Future Biopharmaceutical Human Resources Training and Research Team, Hanyang University, Seoul, 04763 Republic of Korea.
J Pharm Investig. 2018;48(2):199-208. doi: 10.1007/s40005-017-0381-1. Epub 2017 Dec 28.
Cells naturally sense and actively response to their environment. Cell-therapy has long been studied and shown therapeutic effects in various diseases. However, several hurdles should be overcome to improve cell-based therapy. Gene delivery-mediated cellular modification has shown improvement of cell function by obstacle gene silencing and therapeutic gene expression. Especially, CRISPR/Cas9-mediated genome editing is a very promising method for gene modification. In this review, we describe the recent advances in genetic modification for cell therapy. Stem cells are still promising source of cell therapy due to their self-renewal character and differentiation potential. Immune cells regulate the inflammatory response and immunization, which inspired various cell therapy using immune-regulatory cells. Conclusively, we emphasize the need to develop gene-modification-based cell therapy as potent future treatment.
细胞能够自然感知并积极响应其周围环境。细胞疗法长期以来一直被研究，并在各种疾病中显示出治疗效果。然而，为了改进基于细胞的疗法，还需要克服几个障碍。基因递送介导的细胞修饰已通过障碍基因沉默和治疗性基因表达显示出细胞功能的改善。特别是，CRISPR/Cas9介导的基因组编辑是一种非常有前景的基因修饰方法。在这篇综述中，我们描述了细胞治疗基因修饰的最新进展。干细胞由于其自我更新特性和分化潜能，仍然是细胞治疗的有前景的来源。免疫细胞调节炎症反应和免疫，这激发了使用免疫调节细胞的各种细胞治疗。总之，我们强调需要开发基于基因修饰的细胞疗法作为未来有效的治疗方法。
