Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease
| Autor: | Karen Tylee, Trevor F. Carr, Alan Cooper, M. Thornley, Ed Wraith, Anne O'Meara, Jean Mercer, Heather J. Church, Rob Wynn |
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| Rok vydání: | 2007 |
| Předmět: |
medicine.medical_specialty
Heterozygote medicine.medical_treatment Mucopolysaccharidosis I Dermatan Sulfate Histocompatibility Testing Cord Blood Stem Cell Transplantation Hematopoietic stem cell transplantation Chimerism Andrology Iduronidase Internal medicine Medicine Humans Transplantation Homologous Hurler syndrome Glycosaminoglycans Transplantation Hematology business.industry Donor selection Chondroitin Sulfates Hematopoietic Stem Cell Transplantation medicine.disease Treatment Outcome Immunology Stem cell business |
| Zdroj: | Bone marrow transplantation. 39(4) |
| ISSN: | 0268-3369 |
| Popis: | Hurler Syndrome is corrected by allogeneic BMT by the action of donor enzyme on recipient tissue. In this paper, we describe monitoring of 39 patients transplanted in two centres to determine donor chimerism, enzyme level and residual substrate - expressed as dermatan sulphate to chondroitin sulphate ratio. We show that in fully engrafted recipients, the enzyme level, expressed as mumol/g total protein/h, post-transplant is 24.2 from an unrelated donor and 10.2 from a heterozygote family donor (P |
| Databáze: | OpenAIRE |
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