A CRISPR-based fix for human sickle cells shows promise in mice

(By Sharon Begley for STAT)

Scientists have taken an important step toward using CRISPR-Cas9 genome-editing to cure sickle cell disease1, repairing the disease-causing mutation in blood-forming cells taken from patients. Some edited cells, injected into lab mice, both survived in the animals’ bone marrow and turned into red blood cells — a hint that CRISPR’d cells would  would produce healthy hemoglobin in people. This is the first such experiment to get levels of healthy hemoglobin that might be high enough to cure patients. Continue reading article here……


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