Reference no: EM133360872
Question: Sickle cell anemia is a genetic disease that affects hemoglobin production. It results from a single nucleotide change in the hemoglobin gene that causes an amino acid substitution in the hemoglobin protein from glutamic acid to valine. The resulting proteins stick together to form long fibers, causing the development of irregular, crescent-shaped red blood cells (RBCs). The sickle cells die early, which causes a constant shortage of RBCs, and, as a result, insufficient oxygen delivery to tissues. In addition, sickled RBCs circulate through small blood vessels, they get stuck and clog the blood flow.
The result is painful episodes of fatigue and abdominal and bone pain, all hallmarks of the condition. Several approaches for treatment of sickle cell anemia have been developed, several involving the use of the gene editing technique, CRISPR. In these treatments, hematopoietic (blood cell forming) stem cells are extracted from the patient, corrected, and then replaced.
In one approach, CRISPR is used to correct the disease-causing mutation in the hemoglobin gene of the isolated hematopoietic stem cells. In another, CRISPR is used to inactivate (knock-out) a gene that normally suppresses expression of fetal hemoglobin after birth. This results in re-activation of fetal hemoglobin expression in the adult.
Which of these two approaches would you expect to better ameliorate the symptoms of sickle cell anemia. Provide two reasons for your choice.
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