Sickle cell anemia is a disease that can be inherited when an individual has parents that carry the sickle cell trait. The sickle cell gene is shown more often in people that are from Middle Easter, Indian, Mediterranean and African descent. In fact, today in America 8 in 10 African-Americans have sickle cell anemia. The gene is a result of malaria being prominent in those specific regions of the world and now in America individuals who are affected are still dying as early as their 40s from its detrimental effects. However, physicians have begun to realized that there are cases where a child’s parents were both carriers of the sickle cell trait, but do not develop the symptoms of the deadly disease. This is because along with the mutation one may develop an addition mutation gene that counteracts sickle cell. So now researchers are determining the best method to mock the same mutation through gene-engineering to help individuals who suffer from sickle cell anemia.
The problem that cause sickle cell anemia’s symptoms is the shape of the molecules within the red blood cells. The pointed shape punctures the red blood cell as the sickle attach to one another when there is a lack of oxygen in the body. This then leads to the red blood cells having a shorter life span and the process of regenerating new cells is too slow. As a result, the person body may experience inflammation.
One method being explored to help relieve individuals of sickle cell disease is turning off the gene that controls the release of the deformed cells. A researcher named Stuart Orkin and his team have recently found the spot on the DNA sequence that produces the fetal hemoglobin. In order to stop the production of these blood cells the researchers test gene-editing by using a zinc scissors to cut the necessary to remove the spot on the DNA sequence. Other researchers and companies are taking another approach to solve the daunting task by administering virus to trigger stems cells that can produce healthy blood cells. Although there is evidence that these procedures work, there are consequences that come along with them. Patients who undergo either procedure have to endure chemotherapy to wipe out the existing stem cells that produce the fetal hemoglobin. This leads to the development of cancer in individuals after several years of treatment and infertility.
Alternatives to these methods are underway that will allow patients to have the same gene therapy treatment without the painful and long-lasting side effects. The Global Blood Therapeutic in the form of a pill is developing one alternative. The pill will allow the hemoglobin to remain distant from oxygen so that the blood cells do not stick together. Although there are many risks that come along with finding a treatment for patients who suffer from sickle cell anemia it is worth exploring the different avenues so that medicine advances. One way to possibly combat the side effects of chemotherapy is to keep the treatment to a minimum and see if a combination of the treatments can be used to compensate for less chemotherapy.