Outcome of Hematopoietic Stem Cell Transplantation in Wiskott-Aldrich Syndrome
نویسندگان
چکیده
منابع مشابه
[Hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome].
BACKGROUND Wiskott-Aldrich syndrome (WAS) is an X linked congenital disease that presents as eczema, thrombocytopenia and immune deficiency. The only curative procedure for this illness is hematopoietic stem cell transplant (HSCT), preferably from a healthy HLA identical sibling donor. Cord blood is becoming an excellent alternative as stem cell source from unrelated donors. AIM To report our...
متن کاملBrief report Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation
Allogeneic hematopoietic stem cell transplantation (HSCT) corrects the WiskottAldrich syndrome (WAS) phenotype. However, the toxicity and mortality frequently associated with this approach warrant the exploration of new therapeutic strategies. Transplantation studies of a murine model of WAS deficiency have been limited by the occurrence of a radiationinduced fatal exacerbation of a preexisting...
متن کاملCorrection of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation.
Allogeneic hematopoietic stem cell transplantation (HSCT) corrects the Wiskott-Aldrich syndrome (WAS) phenotype. However, the toxicity and mortality frequently associated with this approach warrant 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-exist...
متن کاملPediatric Hematopoietic Stem Cell Transplantation
The introduction and evolution of hematopoietic stem cell transplantation (HSCT) could be traced back to 1950s, to the studies on interactions among irradiation, covering spleen and bone marrow from it and injection of bone marrow cells. Today, HSCT is considered a well-established, effective and promising means of therapy for various malignant and non-malignant medical conditions, both in chil...
متن کاملLentiviral hematopoietic stem cell gene therapy in patients with wiskott-Aldrich syndrome.
Wiskott-Aldrich syndrome (WAS) is an inherited immunodeficiency caused by mutations in the gene encoding WASP, a protein regulating the cytoskeleton. Hematopoietic stem/progenitor cell (HSPC) transplants can be curative, but, when matched donors are unavailable, infusion of autologous HSPCs modified ex vivo by gene therapy is an alternative approach. We used a lentiviral vector encoding functio...
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ژورنال
عنوان ژورنال: Clinical Pediatric Hematology-Oncology
سال: 2018
ISSN: 2233-5250,2233-4580
DOI: 10.15264/cpho.2018.25.2.149