US approves world’s first CRISPR gene editing drug
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The FDA, the US Food and Drug Administration, has approved the world’s first CRISPR gene editing drug for the treatment of sickle cell anemia, Casgevy.
The Wall Street Journal called This is a “landmark decision” that will provide a new type of drug that can combat genetic diseases that are very difficult to treat.
Sickle cell anemia is a hereditary disease in which the hemoglobin protein acquires a special crystalline structure. Carriers of the disease during hypoxia, for example when climbing a mountain, may experience symptoms such as anemia, attacks of pain, swelling and inflammation of the fingers or toes, blood thrombosis in the spleen and liver, damage to the lungs and heart, leg ulcers, avascular necrosis and eye damage.
The drug in question eliminated pain attacks in 95% of the subjects, and not a single patient experienced transplant rejection.
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