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Prepublished online as a Blood First Edition Paper on April 17, 2002; DOI 10.1182/blood-2001-12-0319.
Submitted December 27, 2001
Accepted January 30, 2002
Correction of the murine Wiskott-Aldrich syndrome (WAS) phenotype by hematopoietic stem cell transplantation
Ted S Strom, Xiuling Li, John M Cunningham, and Arthur W Nienhuis*
Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, TN, USA; Pathology, St. Jude Children's Research Hospital, Memphis, TN, USA
Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, TN, USA
Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, TN, USA; Biochemistry and Pediatrics, University of Tennessee, Memphis, TN, USA
* Corresponding author; email: arthur.nienhuis{at}stjude.org.
Allogeneic hematopoietic stem cell transplantation (HSCT) corrects the Wiskott-Aldrich syndrome (WAS) phenotype. However, the toxicity and mortality frequently associated with this approach warrants the exploration of new therapeutic strategies. Transplantation studies of a murine model of WAS deficiency have been limited by the occurrence of a radiation-induced fatal exacerbation of a pre-existing colitis in the peri-transplant period. Here we demonstrate that when crossed to a C57/B6 background, WAS-deficient males show little if any colitis and reliably survive HSCT. We show that HSCT corrects the hematologic and functional deficiencies of WAS KO mice. These results strengthen the analogy between murine and human WAS, and provide a basis for the use of WAS deficient mice to explore novel approaches to correction of the disease phenotype.
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