Gene Therapy and CRISPR: Treating Genetic Diseases

DNA is made up of millions of base pairs. Genes make up everything about living organisms. Any defect in genes can cause a variety of issues and contribute to diseases. CRISPR is commonly used in gene therapy, a branch of healthcare that concentrates on changing a person’s genes to treat a genetic disease. The U.S. Food and Drug Administration (FDA) regulates the process of testing and approving gene editing therapy. The FDA must give approval in order for a researcher to attempt a clinical study. The organization has every right to reject any clinical trial that is seen as a potential threat to the people participating in the study. There are several occasions where gene therapy proved to be successful, one being the use of CRISPR on patients who suffer from sickle cell disease and Beta-thalassemia. Millions of people globally suffer from sickle cell, a genetic disease resulting in the creation of crescent-shaped red blood cells that are more sticky and stiff than regular cells. This can lead to anemia and other potentially fatal health issues. Beta thalassemia is a different mutation that produces less hemoglobin, which allows red blood cells to carry oxygen. For individuals who can find compatible donors, bone marrow transplants could be a possible solution. Sickle cell disease and Beta thalassemia are the results of heritable, single-cell mutations making these two diseases excellent candidates for gene editing therapies. During a clinical trial, Beta thalassemia and sickle cell patients were treated with CTXoo1, a CRISPR therapy. The treatment was that a gene was delivered to boost the production of healthy, normal hemoglobin.The 15 patients with Beta thalassemia all had higher hemoglobin levels after about three months. The patients who received this treatment no longer needed blood transfusions. The seven sickle cell patients received the exact same care, and after three months they reported increased hemoglobin levels without experiencing any discomfort.