Article
Expression of human Wiskott-Aldrich syndrome protein in patients' cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins.
Gene therapy - 1 Feb 2000
Huang M M, Tsuboi S, Wong A, Yu X J, Oh-Eda M, Derry J M, Francke U, Fukuda M, Weinberg K I, Kohn D B
Abstract excerpt
The Wiskott-Aldrich syndrome (WAS) is an uncommon X-linked recessive disease characterized by thrombocytopenia, eczema and immunodeficiency. The biochemical defect of this disorder primarily affects cells derived from bone marrow. To understand better the molecular mechanisms underlying this disease and to evaluate the possibility of correcting the genetic defects in hematopoietic cells, a Moloney murine leukemia...
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