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X-SCIDX-Linked Severe Combined Immunodeficiency
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On a quest for a highly selective immunosuppressive drug, researchers from Pfizer sought to learn from a human immune disorder: X-chromosome-linked severe combined immunodeficiency, or X-SCID, which is characterized by massively dysfunctional immune cells (both B and T cells).
Seven children and one adult have been treated for X-SCID at Great Ormond Street.
Allain Fischer of the Hopital Necker-Enfants Malades, Paris, and colleagues who previously reported successful gene therapy results in four infants with X-SCID.