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Genomics Miracles

Updated: Apr 24, 2021

By Aila (with help from Lihong)

Genome sequencing and gene editing are saving lives. Genome sequencing helps to identify gene related diseases, viruses, and disorders. In this post I share stories about how gene therapy cures people that have rare genetic disorders or protect people from viruses that have recently just come about. The three that I will be sharing with you today will be Sickle Cell, Covid-19, and Spinal muscular atrophy (SMA). These illnesses may seem to be very different but they all use similar tools to treat or cure. To learn more read this article.

Sickle cell is a disease that affects your blood cells. This disease can cause extreme amounts of pain and can often be fatal. It is caused by a mutation that turns blood cells into the shape of a sickle. Blood cells carry oxygen to different places of the body, so when you have sickle cell your body is not able to get enough oxygen. When you have sickle cell you have to get your blood drained out and then you get new blood put back into you every few months. Sometimes if it is too extreme you might have to get a bone marrow transplant that is extremely painful and still does not come close to completely curing this disease. Thanks to gene therapy, there is a potential cure for sickle cell that is less painful and almost completely cures the disease with one treatment. Approximately 100,000 Americans currently have sickle cell. Many patients under the clinical tr