Results from a clinical trial have demonstrated that transplantation of hematopoietic stem and progenitor cells with CCR5 knockdown into patients with HIV-1 infection and acute lymphoblastic leukemia by CRISPR editing can achieve a long-term reconstitution of the patient’s hematopoietic system without the occurrence of gene editing-related side effects, and can produce partial control of HIV infection during ART cessation. This evidence concerns the gene CCR5 and HIV infectious disease.