![]() ![]() In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. Beta thalassemia is a blood disorder that reduces the production of hemoglobin - the iron-containing protein in red blood cells that carries oxygen to cells throughout the body. The disease can cause extreme pain and damage the lungs, heart, kidneys and liver. These cells can lyse and obstruct small blood vessels, depriving the body's tissues of oxygen. Sickle cell disease is an inherited defect of the hemoglobin that causes the red blood cells to become crescent-shaped. Also, the gene manipulation does not use a viral vector as with other gene therapy studies but is done with electroporation (quick production of pores into the cells with high voltage) which is known to have low risk of off-target gene activation, according to Rondelli. The advantage of this approach is that it uses the patient's cells with no need for a donor. By doing so, stem cells start producing fetal hemoglobin so that patients with congenital hemoglobin defects (beta thalassemia or sickle cell disease) make enough fetal hemoglobin to overcome the effect of the defective hemoglobin that causes their disease. In the paper published in the New England Journal of Medicine, CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia, researchers reported gene editing modified the DNA of stem cells by deleting the gene BCL11A, the gene responsible for suppressing fetal hemoglobin production. The two researchers who invented this technology received the Nobel Prize in Chemistry in 2020. The article reports two patients have been cured of beta thalassemia and sickle cell disease after their own genes were edited with CRISPR-Cas9 technology. Damiano Rondelli, the Michael Reese Professor of Hematology at the UIC College of Medicine. The first cases treated with this approach were recently published in an article co-authored by Dr. ![]()
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