Question

L-glutamine powder was approved to treat this disease in 2017. bluebird bio’s lentiviral treatment for this disease was approved on the same day in November 2023 as the first-ever CRISPR gene therapy, (20[1])which (20[1])also treats this condition. (20[7])For 50 years, the (20[1])only available drug for this molecular disease was a derivative of urea that activates synthesis of an alpha-gamma tetramer (20[1])normally made only by (*) infants. This disease can be treated by transplanting bone marrow (10[1])without the glutamate-to-valine (10[2])mutation (10[1])in beta-globin. (10[7])Sufferers of this disease experience excruciating (10[1])vaso-occlusion (10[1])crises when erythrocytes get stuck in capillaries. (10[1])For 10 points, name (10[1])this genetic disease common (10[1])in Black Americans which deforms (10[1])red blood cells. (10[1])■END■ (10[7])

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