Maryam Behfar

From oncogenesis to approval: the lentiviral gene therapy revolution in Wiskott–Aldrich syndrome

Wiskott–Aldrich syndrome (WAS) is an X-linked primary immunodeficiency caused by pathogenic variants in the WAS gene, resulting in absent or dysfunctional WAS protein (WASp) and a clinical triad of eczema, recurrent infections, and micro-thrombocytopenia. Allogeneic hematopoietic...