Gene Therapy As An Efficient Way To Cure Hemophilia

Hemophilia is a bleeding disease which causes strong or abnormal bleeding and weak blood clotting. This disorder is inherited through a genetic pattern from both parents, females carry the disease and are rarely affected by it and males have a greater chance of being influenced by hemophilia. There are two types of hemophilia, A and B. Hemophilia A is a shortage of Factor VIII, Factor VIII is a blood clotting protein, it is also called anti-hemophilia factor (AHF). Hemophilia B has deficiency of Factor IX, it is also called ‘Christmas Disease’ because it was first identified by a patient called Stephen Christmas. Factor IX is a protein that produces blood clots. The symptoms of Hemophilia include abnormal bleeding from anywhere of your body and long-term damage in your joints from bleeding in joint spaces which causes pain, swelling and developing deformities. Hemophilia B has a subgroup called Leyden Phenotype which affects children but improves during puberty. In the world hemophilia A is more common and occurs in 1 out of 5000 male births and Hemophilia appears in 1 out of 20000 to 30000 people. It is very rare for women to suffer from Hemophilia however it does happen.

The most effective way to treat Hemophilia would be through gene therapy since it is a gene related disorder. By having gene therapy patients are able to lead a fairly normal life. For gene therapy in Hemophilia B, the missing gene of the blood clotting protein Factor IX is added to a special engineered virus which travels to the liver and then transfers it to the gene. This leads to the patient hardly ever bleeding. Gene therapy in Hemophilia A is not as advanced as in Hemophilia B but recent studies in the United Kingdoms have had successful outcomes. Would be patients having almost the same values of clotting protein as people without Hemophilia.

As for Hemophilia A, they use a virus to deliver the gene of the protein Factor VIII which was also altered by deleting a certain part to fit the coding better into the viral vector. There were three different levels of protein doses by having low, medium and high dose groups. Observations indicate that patients with a higher dose had similar Factor VIII levels as for people with a normal protein level for a year after the treatment. The positive social effects for gene therapy would be being able to do numerous activities without getting a wound and starting to bleed. Since many patients with hemophilia are children, this restricts them from playing sports and being able to socialize with their friends. This also often interferes with the development of the mind and physical body. Examples of such impacts be seen by independence and risk-taking activities. Without such activities the patients are not able to take care of challenging situations properly later on in life. Since gene therapy sessions are not as time consuming in the longer run as current treatment techniques, patients especially children would be able to interact and at some point would be able to play sports or other physical activities without getting hurt. When children are playing they are testing their own boundaries and taking part in risky activities like climbing trees or rocks which leads them to finding their own boundaries and developing skills such as perception and competence. They are also able to learn and improve in activities which require lots of practice and challenging yourself.

A negative impact of gene therapy would be how high the cost is. The price range for gene therapy in hemophilia can be from 1.5 to 2 million dollars per patients. Such high prices could have to do with gene therapy being a one-time cure and the medicine companies who have invested in it want to be able to get some kind of profit. If gene therapy would be cheaper more people would be able to afford such treatment. This would lead to medicine companies going out of business since no one needs their medicine anymore but also the companies are not making enough money through gene therapy because the patients will only need it once. Other current treatments cost 1 million dollar per year per patient but the government cover 30 to 40 percent. The health insurance does not cover for gene therapy since it is still uncertain if it will work for every patient, it is considered the first 1 million dollar drug. In developing countries such as in Africa, gene therapy would be very helpful to cure hemophilia, other genetic diseases but also in viruses such as Ebola, HIV-1 and hepatitis B. There are not enough researchers for such treatments since there is not a constant state delivery of equipment and material because there is not a proper support of money. Also the current treatment options for hemophilia are very expensive for patients and there are no proper health care systems because the economy is not stable and there is not continuous progress.

I conclude that gene therapy could become a very efficient way to cure hemophilia. However since it is still uncertain if this therapy will always work for every patient and for Hemophilia A patients it is not advanced enough to be used as treatment. If the gene therapy would be used as a cure then this would have major social benefits for the younger patients. Since they would be able to interact with others and develop important future life skills such as being risk-taker without interference. The main negative impact of gene therapy would be that it is very expensive which would mean that average citizens would not be able to afford this treatment. In developing countries this would be a major issue since they often aren’t even able to afford the current, existing treatments. So far there is no guarantee that gene therapy will work for all hemophilia patients meaning that investing in this gene therapy would not be worth the money.

03 December 2019
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