Hemophilia B: Genes in Hemophilia B, Diagnosis, and Treament Approach Using Adeno-Associated Virus Vectors

Abstract

Background: Hemophilia B is a hemorrhage disorder inherited based on the X-linked inheritance pattern. Diagnosis of this disease can include such as fetal DNA test. Adeno-associated virus (AAV) delivery vehicles can be used for treating hemophilia B. In this study, the author reports the progress in a study of hemophilia B focused on genetic inheritance and treatment of the disease with AAV delivery vehicles as objectives of this study. Methods: The author searched Google, ScienceDirect and the PubMed Database at NCBI for articles on hemophilia B and treatment of hemophilia B with AAV vectors . Results: Mutations in the F9 gene result in hemophilia B. Diagnosis of hemophilia B can include such as fetal DNA and cell-free fetal DNA tests. Currently, an additional plasma-derived or recombinant factor IX are drugs for treating this disease. Unfortunately, this approach can permanently not recover patients with hemophilia B. Therefore, it needs another approach for treating this disease such as AAV vectors. Research results in animal model have demonstrated great promise for treatment of hemophilia B with these vectors. Conclusions: Hemophilia B is inherited according to X-linked pattern. To detect this disorder, cell-free fetal DNA test, for example, can be used. Adeno-associated virus vectors are a helpful tool for treatment of hemophilia B.