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.
Researchers optimize gene editing for SCD and beta thalassemia
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..
