Niemann–Pick disease type C (a cellular cholesterol lipidosis) treated by bone marrow transplantation
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چکیده
منابع مشابه
Intractable diseases treated with intra-bone marrow-bone marrow transplantation
Bone marrow transplantation (BMT) is used to treat hematological disorders, autoimmune diseases (ADs) and lymphoid cancers. Intra bone marrow-BMT (IBM-BMT) has been proven to be a powerful strategy for allogeneic BMT due to the rapid hematopoietic recovery and the complete restoration of T cell functions. IBM-BMT not only replaces hematopoietic stem cells (HSCs) but also mesenchymal stromal cel...
متن کاملCongenital erythropoietic porphyria successfully treated by allogeneic bone marrow transplantation.
The long-term biochemical and clinical effectiveness of allogenic bone marrow transplantation (BMT) was shown in a severely affected, transfusion-dependent 18-month-old female with congenital erythropoietic porphyria (CEP), an autosomal recessive inborn error of heme biosynthesis resulting from mutations in the uroporphyrinogen III synthase (URO-synthase) gene. Three years post-BMT, the recipie...
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Eighteen patients, twelve men and six women, with aplastic anemia underwent allogenic bone marrow transplantation (BMT) from HLA-matched siblings during the period of 1990 to 1996. The conditioning regimen was cyclophosphamide with or without busulfan, depending on the cause of aplasia. Antilymphocyte globulin (ALG) and cyclosporine were used for rejection and acute GVHD prophylaxis, respe...
متن کاملBone Marrow Transplantation in Thalassemia (Part 2)
During the last two decades conventional therapy has improved the prognosis of thalassemia. However, despite such improvement it still remains a progressive disease with treatment-related complications such as hepatitis, liver fibrosis, and cardiac disease. Bone marrow transplantation (BMT) can prevent or delay progression of the aforementioned complications. The importance of clinical research...
متن کاملBone Marrow Transplantation in Thalassemia (Part 1)
During the last two decades conventional therapy has improved the prognosis of thalassemia. However, despite such improvement it still remains a progressive disease with treatment-related complications such as hepatitis, liver fibrosis, and cardiac disease. Bone marrow transplantation (BMT) can prevent or delay progression of the aforementioned complications. The importance of clinical research...
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ژورنال
عنوان ژورنال: Bone Marrow Transplantation
سال: 1999
ISSN: 0268-3369,1476-5365
DOI: 10.1038/sj.bmt.1701826