EBMT Global Perspective | Professor Francesca Ferrua: Breakthroughs in Autologous Hematopoietic Stem Cell Gene Therapy for WAS—From Concept to Clinical Practice
Wiskott–Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency characterized by thrombocytopenia, eczema, and recurrent infections, posing a serious threat to affected children. While allogeneic hematopoietic stem cell transplantation (HSCT) can be curative, it is limited by donor availability, the risk of graft-versus-host disease (GVHD), and conditioning-related toxicity.





