Novel therapy improves immune function in teen with rare disease

In a novel approach that works around the gene defect in Wiskott-Aldrich syndrome, an inherited immune deficiency disorder, researchers used an alternative cell signaling pathway to significantly improve immune function in a 13-year-old boy with the disease. The study, at The Children's Hospital of Philadelphia, provides a proof-of-principle that immunotherapy, which harnesses elements of the body's immune system, may be used to treat this rare but often deadly disorder.

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