Combinatorial Strategies for Long-term Control of HIV Infection

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 gene-edited 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.