A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation

Wiskott-Aldrich syndrome (WAS) is a rare X-linked primary immunodefciency characterized by microthrombocytopenia, recurrent infections, eczema, and increased susceptibility to autoimmunity and tumors. This disease is caused by mutations in the gene encoding for WAS protein (WASp), predominately expressed in hematopoietic cells. WASp alterations can lead to a wide clinical phenotype, ranging from classical to milder forms, depending on the type and severity of the WAS gene mutations