Genome editing-based HIV therapies

• A therapy beyond highly-active antiretroviral therapy is needed.
• Genome editing-based therapy has achieved significant progress.
• Recent clinical trial results are exciting although some problems exist.
• Long-term evaluation of safety and efficacy is required.

Genome editing (GE)-based HIV therapy is achieved by modification of infection-related genes to produce HIV-resistant cells followed by reinfusion of the modified cells into patients. The ultimate goal is to achieve a functional or actual cure for HIV infection. Despite multiple potential targets for GE-based HIV therapies, CCR5 is the most feasible owing to the naturally existing CCR5 δ32 genotype which confers resistance to HIV. A recent clinical trial of infusion of modified autologous CD4+ T cells proved safety and efficacy within the limits of the studies. However, long-term evaluation of the safety and efficacy is required before GE-based HIV therapy is ready for clinical implementation.