Wiskott-Aldrich syndrome: Long-term results of gene therapy developed

Дата публикации: 24-09-2026 21:20:12

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 affected children from an early age to severe infections, bleeding episodes, eczema and autoimmune complications.

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