Curing Congenital Diseases?
In 2020, the Nobel Prize was awarded to two chemists who invented a gene editing technology. That technology, CRISPR-Cas9, was recently used successfully on two patients with beta thalassemia and sickle cell disease. The New England Journal of Medicine reports that “gene editing modified the DNA of stem cells by deleting the gene BCL11A, the gene responsible for suppressing fetal hemoglobin production. 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.”
Article coauthor Dr. Damiano Rondelli, the Michael Reese Professor of Hematology at the University of Illinois Chicago (UIC) College of Medicine, commented, “UIC and UI Health is an ideal place for any cellular therapy in sickle cell disease because of our experience and success in stem cell transplantation in these patients. In fact, over 75% of sickle cell patients can be cured with a transplant, and we have already done over 50 cases.” The research presented is “game-changing” for those affected by these life-threatening monogenic diseases. The hope for the future: trials offering participants the ability to send their cells in to undergo editing; then, after chemotherapy, patient’s will have their edited stem cells reinserted into their bloodstream.
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