Sickle Cell anemia is not a name that people don’t know of. It is a disease that has been around since 1910 and continues to affect people internationally. All we have had in human history are quick fixes for it. Now, there may be hope for victims in the form of gene therapy.
Sickle Cell Anemia: What is it and what does it do?
Sickle cell anemia is a hereditary disease that affects up to 100 million people internationally. Victims of it do not have enough healthy blood cells to carry oxygen to their bodies. A healthy person had red, flexible, and circular blood cells. The kind that people picture when the term “blood cell” is mentioned. Sickle cell anemia, however, results in the blood cells taking on a crescent, shrunken shape similar to its namesake. They are rigid resulting in extensive pain to the patient.
The Solution in Place
The current treatment for the disease is a blood transfusion. As stated in the name, blood transfusions are the transfer of blood from the donor to the recipient. It is performed by attaching a small tube to both people. This practice is used for life-threatening injuries in which someone has lost massive amounts of blood as well. There are specific conditions for when this can occur though since the blood of the recipient and donor must be compatible. This is used for those who suffer from sickle cell anemia since healthy blood cells are donated in order to make up for the unhealthy cells. Unfortunately, this is only used to manage the disease, not cure it completely.
Gene therapy is a practice that medical professionals use to fix defective genes to cure fatal diseases. Such diseases can include cancer, heart disease, diabetes, and AIDS. While promising, this treatment does have some negatives. The carrier viruses that house the new genes can affect more than the intended target. There could be a misplacement of the virus and that could result in unwanted side effects which could prove fatal. This risk factor is very low and the positives outweigh the negatives by a large margin. This treatment can and has helped many people, with approximately 2,335 clinics established since 2015.
Gene Therapy vs. Sickle Cell Anemia?
Sickle Cell anemia is, as previously stated, a hereditary disease. This means that there is a specific gene in victims’ DNA that results in the production of rigid, sickle cells. Through the use of Gene therapy, that gene could be replaced with a healthy blood cell gene. This could do more than help the immediate patient, but prevent future generations from experiencing the excruciating pain it brings along with it. The two specific treatments are CRISPR–Cas9 genome editing and coding a strand of RNA. The CRISPR-Cas9 genome editing method is the more promising one out of the two, as it is the first published treatment used to cure heritable diseases. RNA coding is a solution as well, rewriting the harmful mutation of the hemoglobin gene.
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