Recombinant lentivirus vector for treating beta-globulin afunction, preparation method and application

Abstract

The application discloses a recombinant lentivirus vector for treating beta-globulin afunction, a preparation method and application. A gene element of the recombinant lentivirus vector disclosed by the application is composed of a left side lentivirus long terminal repeat, a lentivirus counter-response element, a center poly-purine sequence, a gene locus regulating and controlling sequence of a beta-globulin gene, a beta-globulin gene, of which 87-th threonine is mutated into glutamine, and a right side lentivirus long terminal repeat which are connected in order. According to the recombinant lentivirus vector disclosed by the application, through optimizing and improving the gene element, the gene transduction efficiency is increased, and the beta-globulin gene can be efficiently expressed during erythroid differentiation of hemopoietic stem cells, so that the consumption of viruses can be effectively lowered, and the treatment cost is reduced while the safety is improved. The recombinant lentivirus vector disclosed by the application provides a novel treatment tool and scheme for beta-thalassemia and sickle cell anemia caused by the beta-globulin afunction.

Description

technical field [0001] The present application relates to the field of treatment of beta-globin function loss diseases, in particular to a recombinant lentiviral vector for treating beta-globin function loss, its preparation method and application. Background technique [0002] Mutations in the β-globin gene (HBB) can cause serious genetic diseases, including β-thalassemia caused by loss of gene function and sickle cell anemia caused by a point mutation of the sixth amino acid site Glu to Val. Mutations in the HBB gene affect the production of β-globulin, resulting in an imbalance between α-globulin and β-globulin in red blood cells, as well as abnormal hemoglobin, which in turn leads to erythropoietic disorders and hemolytic anemia. [0003] At present, the conventional treatment for patients with transfusion-resistant β-thalassemia major (TDT) is regular blood transfusion and adequate iron removal therapy. Based on a thalassemia patient's life expectancy of 50 years, the ...

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Problems solved by technology

Moreover, the BB305 virus may have complex production processes and high costs due to the structure of the virus, and the price of the final product is as high as 1.8 million US dollars

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