Wednesday, December 1, 2021

Gene Therapies Used to Treat Sickle Cell Anemia and B-thalassaemia

Sickle cell anemia and B-thalassaemia are two genetic blood disorders that are caused by the mutation of the B-globin gene. Sickle cell anemia (SCA) is a form of anemia where the redblood cells are sickle, or crescent moon shaped. B-thalassaemia is a form of anemia that causes a reduction in the production of hemoglobin. Both of these forms of anemia affect the way that red blood cells contain and carry oxygen, which negatively influences our physiology and overall quality of life. Although the only form of treatment for these types of anemia are stem cell/bone marrow transplants, this article highlights gene therapy techniques including CRISPR-Cas9 as well as using RNA to alter the B-globin gene have been used to treat the painful symptoms, otherwise known as pain crisis that follow SCA and B-thalassaemia. These gene therapies were used to change the expression of the B-globin gene that affects these anemias at the level of fetal development and turns them off right after birth. In doing this,it was hypothesized that more -globin could be produced.  The CRISPR-Cas9 approach altered the expression of the BCL11A gene in red blood cells while the RNA approach copied the functioning B-globin gene and placed it into blood-producing stem cells. The downsides to these therapies are issues in fertility, abdominal pain and infection. These therapies are still being tested to further improve the treatment of these therapies.

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