From the National Institute of Allergy and Infectious Diseases:

NIH Scientists Repair Gene Defect in Stem Cells from Patients with Rare Immunodeficiency
CRISPR Gene-Repair Strategy Shows Promise for Treatment of Blood Disorders

Scientists have developed a new approach to repair a defective gene in blood-forming stem cells from patients with a rare genetic immunodeficiency disorder called X-linked chronic granulomatous disease (X-CGD). After transplant into mice, the repaired stem cells developed into normally functioning white blood cells, suggesting the strategy could potentially be used to treat people with this disease.

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