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Two teams target BCL11A in different ways to treat sickle cell disease


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Journal
The New England Journal of Medicine
Reuters Health - 07/12/2020 - Separate teams of doctors using related gene-therapy techniques are reporting positive results in their early-stage efforts to cure severe sickle cell disease, the genetic defect that turns red blood cells misshapen and brittle. Both techniques turn down the activity of a gene known as BCL11A, which suppresses production of the type of hemoglobin that fetuses make. The result: fetal hemoglobin, which doesn't carry the defect that makes adult red blood cells sickle, makes a comeback, reducing or eliminating symptoms. One technique uses a manipulated virus and microRNA to suppress BCL11A. The other employs CRISPR-Cas9 gene editing technology to switch off the gene. Both require patients to receive a bone-marrow transplant of their own manipulated cells. All six patients whose blood was treated with the deactivated virus remain healthy with reduced symptoms or no symptoms after seven to 29 months. One 33-year-old woman with sickle c...


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