US Food and Drug Administration (FDA) approved using CRISPR gene editing therapy to treat sickle cell disease in patients over 12 years of age. Approval was given to two drugs – Casgevy and Lyfgenia. Approval came after studies involving 44 patients with the disease.
Sickle cell anemia is a hereditary genetic abnormality in the structure of hemoglobin (an oxygen-carrying protein found in red blood cells). It is characterized by the appearance of sickle-shaped red blood cells and chronic anemia, which is caused by the destruction of red blood cells. The disease mainly affects black people.
The CRISPR editing technology was developed by American biologist Jennifer Doudna. In 2020, she, along with the Frenchwoman Emmanuelle Charpentier, received the Nobel Prize for this. Called CRISPR-Cas9, or “genetic scissors,” this genome editing technique allows scientists to remove and add pieces of genetic material. It is used to treat hereditary diseases. The UK was the first to approve CRISPR-based therapy.
Details are in the Kommersant article “Whoever fixes DNA will change the world.”
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