Affecting more than 100,000 people in the US, SCD, or sickle cell disease, is an inherited condition that causes a person’s blood cells to block blood flow to the rest of the body. In extreme cases, this disease can cause strokes, eye problems, and many other severe adverse effects in somebody with the illness. As of now, the leading treatment is medication; however, this medication can come with side effects such as lower white blood cell levels and platelet count. Recently, though, a ScienceNews article highlighted a new cure for Sickle Cell Disease that was approved by the Food and Drug Administration.
In the article, a CRISPR gene-editing technique is used to cure the disease. The treatment alters the gentic blueprint of the bone marrow that makes blood cells in a patients body. This process uses a patients own cells to defeat Sickle Cell disease by having edited cells make fetal hemoglobin. Fetal hemoglobin, unlike normal hemoglobin, cant be turned sickle and therfore wont clog up blood streams. In a study following people who received this treatment, 29 out of 30 didnt report any pain crises for a year. There are still side effects of this treatment such as increased exposure to cancer due to chemotherapy needed in the bone marrow altering and potentially other undiscovered sideffects. However, the treatment is still relatively new and it is yet to be seen if it can be improved on and it also still may be a better alternative than the current treatments of Sickle cell disease.
Being a carrier for the sickle cell gene myself, I find this research very interesting. Sickle Cell disease has an autosomal recessive pattern which means that the way to express Sickle Cell disease is through getting two of the recessive genes from both of your parents. Therfore somebody who is heterozygous for sickle cell has a higher chance of having a child with sickle cell disease if there partner is either a carrier or has sickle cell disease than somebody who homozygous dominant for not having sickle cell disease. With this topic being so closely related to me it is important that scientists continue to discover and improve on their ways of curing sickle cell disease in the upcoming generations. If you know any information about any other emerging cures for sickle cell disease share them in the comments below!
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