International journal of hematologyWiskott-Aldrich SyndromeHumansGenetic TherapyWiskott-Aldrich Syndrome ProteinGene Editing
Advancements in gene therapy for Wiskott-Aldrich syndrome: from early trials to emerging approaches.
Luana de Mambro, Roberta Sessa Stilhano
Published: 202610.1007/s12185-025-04099-6
Abstract
Wiskott-Aldrich syndrome (WAS) is a rare X-linked recessive disorder characterized by microthrombocytopenia, eczema, recurrent infections, and immune dysregulation, affecting approximately 1 in 100,000 live births. While the disorder was historically…
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