Gene therapy is subdivided into in vivo and ex vivo according to the tactics of delivery of therapeutic constructions. In vivo therapy, a vector containing a therapeutic construction is injected into the patient. In ex vivo therapy, cells are removed from the patient's body, genetically modified, and then returned. In ex vivo therapy for inherited diseases, hematopoietic stem cells are most often subjected to modification. Despite the advantage of in vivo therapy, which consists in the possibility of scale-up of production and convenience of use of ready-to-use drug, therapy based on transplantation of genetically modified hematopoietic stem cells remains relevant for a number of genetic diseases.