Sickle Cell Gene Therapy
In this article, Elizabeth Dugan talks about the huge medical advances that have created treatments to improve quality of life for those with the incurable disease Sickle Cell.
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Sickle Cell disease new treatment
Sickle Cell disease new treatment
by Elizabeth Dugan
by Elizabeth Dugan
Anthropologists who studied humans from thousands of years ago found an interesting anomaly in some human cells; they appear to have a scythe shape. They would name this mutation sickle cell disease. Sickle cell disease is a mutation in a person’s DNA that causes one’s cells to clump together, leading to serious health complications that could result in death in extreme cases. For years, scientists have been looking for a cure and, in the last decade or so, there has been significant progress in gene therapy.
Sickle Cell disease is a tiny mutation that affects many parts of the human body’s biology, all the way down from the DNA to as high as whole-organism systems. A Berkeley University case study on mutations demonstrates that not all mutations are harmful. After all, without certain mutations, the human body would have little defense against certain illnesses or infections. However, the strange and random mix of mutations that cause one’s cells to morph into a scythe shape can have lasting and harmful effects on a person’s quality of life, even leading to death. The mutated configuration of scythe cells can cause them to cramp up veins and blood pathways and result in blood clots, which can cause organs or appendages to die from lack of oxygenated blood or force excess blood back to the brain, causing a stroke. This can be incredibly painful and deadly, as most modern medicine we have, like blood thinners, does not resolve the root issue.
RNA mixing process by Research Gate
Many scientists have tried to come up with cures or preemptive care for this disease but have had little success until recently with gene therapy. Gene therapy stems from genealogy (the study of how genes work). Scientists believe that, since the general origin of the disease is gene mutation, using different gene mutations that can counter the sickle cell sequence may be the solution. The FDA has approved gene addition and or gene editing to treat Sickle Cell Disease, and has found that, while the treatment does not cure Sickle Cell, it drastically improves the chronic symptoms and greatly reduces death rates in those diagnosed with Sickle Cell Disease. This treatment, done by Yale Medicine Hospital, works via bone marrow being donated by volunteers. Then, the cells in the bone marrow are genetically altered by either adding or editing present cells, and are then transplanted to those plagued with Sickle Cell. This treatment has been working wonders, but it is still not effective enough to be considered viable by the public because of its price point. Even after insurance has done most of the coverage, the treatment can cost well over two million dollars.
Modern science has brought about effective medication in managing Sickle Cell symptoms, but the public has yet to feel the hard work of these scientists, as the price is out of their reach. In the future, the focus is currently working on making gene therapy more financially available to those who need it.
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