Article
Retrovirus-mediated WASP gene transfer corrects defective actin polymerization in B cell lines from Wiskott-Aldrich syndrome patients carrying 'null' mutations.
Gene therapy - 1 Jun 1999
Candotti F, Facchetti F, Blanzuoli L, Stewart D M, Nelson D L, Blaese R M
Abstract excerpt
Boys affected with Wiskott-Aldrich syndrome (WAS) present with variable association of thrombocytopenia, eczema and immune deficiency. If untreated, WAS patients may succumb to intracerebral hemorrhages, severe infections or malignancies. Allogeneic bone marrow transplantation (BMT) can cure all aspects of the disease, but HLA-identical donors are not available to all patients and mismatched BMTs are...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.