GENE THERAPY FOR HAEMOPHILIA

The blood disorder, Hemophilia, has its roots embedded deep into the history of genetic disorders. The European royal family is one of the most prominent families to be affected by this disease thus, dubbing it 'the royal disease'. The types of Hemophilia are divided into two based on the type of coagulation factor mutation found in the patient. For treating haemophilia, gene therapy is done by using different vectors such as lentiviral and retroviral vectors but due to the production of limited expression different adeno associated virus (AAV) strains are used. Some engineerly modified vectors are currently used to get the best possible results. The clinical trials prove the efficacy of these vectors so through their obtained statistical consideration, patient experience and population study once can design vaccines and drugs for haemophilia patients but also due to pre-existing Nabs and pre-existing HCV or HBV infection, the general application of AAV gene therapy is currently limited. The possibility of gene editing for the repair of the mutation is on the horizon.