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Cell Mol Life Sci. 1998 Oct;54(10):1145-53.

Understanding the molecular basis of Wiskott-Aldrich syndrome.

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  • Onyx Pharmaceuticals, Richmond, California 94806, USA. arie@onyx-pharm.com


Wiskott-Aldrich syndrome (WAS) is an X-linked immunodeficiency disorder associated with lymphocytes and platelet abnormalities. The gene that encodes the Wiskott-Aldrich protein (WASP) was recently isolated, and shown to be defective in WAS patients. WASP contains multiple domains that interact with various signalling proteins, including the guanine triphosphatase (GTPase) Cdc42Hs and SH3 domain-containing proteins. Biochemical and genetic evidence strongly suggests that WASP is an important protein in the regulation of cell morphology. Recent progress in the identification of molecular partners for WASP suggests a molecular mechanism for the cellular abnormalities of WAS.

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