Stem Cells and Regenerative Bioengineering Institute in Kangstem Biotech, Gwangmyeong SK TechnoPark, Gyeonggi-do, Seoul, South Korea.
Stem Cells and Regenerative Bioengineering Institute in Kangstem Biotech, Gwangmyeong SK TechnoPark, Gyeonggi-do; Adult Stem Cell Research Center, College of Veterinary Medicine. Seoul National University. Seoul, South Korea.
AIDS Rev. 2020 Dec 23;22(4):175-182. doi: 10.24875/AIDSRev.19000128.
AIDS is a disease caused by a chronic infection of HIV. Recently, long-term control of HIV infection has been demonstrated through the bone marrow transplantation of hematopoietic stem cells (HSC), in which the C-C chemokine receptor type 5 (CCR5) gene is mutated innately. However, it is very difficult to obtain CCR5 mutant HSC that match human leukocyte antigen between donor and recipient. To solve this problem, this review will summarize and discuss various reports related to the generation of patient-specific CCR5 geneedited HSC. The fusion of current gene editing (zinc-finger nuclease, transcription activator-like effector nuclease, and clustered regulatory interspaced short palindromic repeats) and cellular reprogramming technology (somatic cell nuclear transfer, induced pluripotent stem cells technology, and direct phenotypic conversion) enables the generation of patient-specific CCR5 edited HSC. These cells can be useful as valuable therapeutic agents for long-term control of HIV-infected patients in the future.
艾滋病是一种由 HIV 慢性感染引起的疾病。最近，通过骨髓移植造血干细胞（HSC）已经证明了 HIV 感染的长期控制，其中 C-C 趋化因子受体 5 型（CCR5）基因先天发生突变。然而，很难在供体和受体之间获得匹配人类白细胞抗原的 CCR5 突变型 HSC。为了解决这个问题，本综述将总结和讨论与生成患者特异性 CCR5 基因编辑 HSC 相关的各种报告。当前基因编辑（锌指核酸酶、转录激活因子样效应核酸酶和簇状规则间隔短回文重复）和细胞重编程技术（体细胞核转移、诱导多能干细胞技术和直接表型转化）的融合使得能够生成患者特异性 CCR5 编辑 HSC。这些细胞将来可能成为治疗 HIV 感染患者的有价值的治疗剂。
