Strategies for Hemophilia Treatment, a literature review of current evidence

Hemophilia is an inherited bleeding disorder caused by malfunctioning or lacking blood coagulation factor VIII (hemophilia A) or IX (hemophilia B). Currently, the main treatments for these X-linked diseases are replacement therapy using periodic and regular injections of plasma-derived coagulation factors or their recombinant products. The use of recombinant coagulation factors is due to the need for periodic and regular injections to prevent bleeding in the acute type of hemophilia and in turn imposing a financial burden on patients. On the other hand, this treatment method is not available to all patients, especially those in developing countries. Recently, gene therapy has been proposed as a suitable, cost-effective, and permanent treatment option for hemophilia. This method is expected to solve the problems we currently face in treating hemophilia. Hemophilia is suitable for gene therapy since an abnormal gene is responsible for the disease. During the last two years, successful clinical trials for gene therapy using vectors derived from adeno-associated virus for both hemophilia A and hemophilia B have been conducted and approved for administration. Moreover, new methods based on gene-cell therapy and genome editing are under investigation for the treatment of hemophilia. This review article deals with different approaches for hemophilia treatments from the past to the present, particularly gene therapy methods.