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Wiskott-Aldrich syndrome: Long-term results of gene therapy developed
A single infusion of genetically corrected autologous hematopoietic stem cells can provide lasting clinical benefits for patients with Wiskott-Aldrich syndrome, a rare genetic disorder that impairs immune and platelet function, exposing…
AI Summary
A single infusion of genetically corrected autologous hematopoietic stem cells can deliver lasting clinical benefits for children with Wiskott‑Aldrich syndrome, a rare disorder that compromises immune and platelet function and leads to early‑onset infections, bleeding, eczema and autoimmune complications.
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