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Chronic Granulomatous Disease Treatment News: NIH Scientists Repair Gene Defect in Stem Cells from Patients with Rare Immunodeficiency

Posted on Posted in Chronic Granulomatous Disease News

(SOURCE: NIH/NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES) – 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 […]