WISKOTT-ALDRICH-SYNDROME - A GENE, A MULTIFUNCTIONAL PROTEIN AND THE BEGINNINGS OF AN EXPLANATION

Patients with Wiskott-Aldrich syndrome show various defects in the nor mal function of platelets and lymphocytes. The recent identification o f the gene responsible for this syndrome has led to a surge of studies aimed at solving the puzzle posed by the varied phenotype observed in this disease. It is now known that WASP, the protein product of this gene, can interact with a large number of other proteins known to be i nvolved in the regulation of signal transduction and cytoskeletal orga nization. Thus, WASP appears to integrate these two basic and fundamen tal cellular mechanisms. Several groups are now focusing on understand ing the function of WASP in detail, and translating this new knowledge into improved therapies.