Gene editing of patients' blood stem cells can potentially cure many blood disorders. But introducing targeted edits into these cells has been challenging, and the edits aren't always stable once the cells engraft in the bone marrow. Researchers now report a CRISPR approach that overcomes these technical challenges. In cell and mouse models of sickle disease and beta thalassemia, they show that edited blood stem cells produce similarly corrected red blood cells making functional hemoglobin.
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Friday, March 29, 2019
Researchers at Dana-Farber/Boston Children's optimize gene editing for SCD and beta thalassemia
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