Advances in gene therapy for hemophilia

Abstract

Hemophilia gene therapy is the process that vectors transfer normal FⅧ and FⅨ gene into patients with hemophilia, in order to correct genetic defects and produce normal FⅧ and FⅨ protein, which can meet the hemostatic requirements and bring new hope for treatment of hemophilia.The transformation of the target gene, as well as how to choose vectors and target cells are important parts of this process. The core technology of hemophilia gene therapy is gene transfer vector technology, and vectors for gene therapy can be divided into non-viral vectors and viral vectors.At present, many researches of gene therapy for hemophilia have made a breakthrough progress. However, hemophilia gene therapy still have some problems, such as the application of the vectors, the selection of target cells, the immune response to the vectors and the expression products, and the like, so further researchs are still needed. Therefore, this article summarizes the gene therapy process and clinical research progress of hemophilia. Key words: Hemophilia; Gene therapy; Genetic vectors; Targeted gene repair