  Hepatocyte transplantation using the domino concept in a child with Tetrabiopterin non-responsive phenylketonuria.; DEBRAY, François-Guillaume  ; et alin Cell Transplantation (2012), 21(12), 2765-70 Phenylketonuria is a metabolic disease caused by phenylalanine hydroxylase deficiency. Treatment is based on a strict natural protein-restricted diet that is associated with the risk of malnutrition and ... [more ▼] Phenylketonuria is a metabolic disease caused by phenylalanine hydroxylase deficiency. Treatment is based on a strict natural protein-restricted diet that is associated with the risk of malnutrition and severe psychosocial burden. Oral administration of tetrahydrobiopterin can increase residual enzyme activity, but most patients with severe clinical phenotypes are non-responders. We performed liver cell transplantation in 6 years-old boy with severe tetrahydrobiopterin non-responsive phenylketonuria, who failed to comply with diet prescriptions. The transplanted hepatocytes were obtained in part from an explanted glycogen storage type 1b liver. Following two infusions, blood phenylalanine levels returned within the therapeutic target while the phenylalanine half-life assessed by loading tests decreased from 43 h to 19 h. However, three months later, blood phenylalanine concentrations increased and the phenylalanine intake had to be reduced. Cell based therapy is a promising therapeutic option in phenylketonuria and the domino concept may solve the issue of cell sources for hepatocyte transplantation. [less ▲] Detailed reference viewed: 13 (5 ULg) Maintenance of functional human cancellous bone and human hematopoiesis in NOD/SCID mice; Humblet, Chantal ; Belaid, Zakia et alin Cell Transplantation (2004), 13(7-8), 823-831 Attempts were made to establish models to study interactions between marrow stromal cells and hematopoietic cells in vivo. The approach was to create a NOD-SCID-hu murine model of long-term human ... [more ▼] Attempts were made to establish models to study interactions between marrow stromal cells and hematopoietic cells in vivo. The approach was to create a NOD-SCID-hu murine model of long-term human hematopoiesis by implantation of a human adult bone fragment. Nine to 12 weeks posuransplantation, human CD45(+) cells were detected in the blood and the spleen of some mice. The histology of the human transplant showed that human bone fragment was viable at 9 weeks. Moreover, vessels of human origin, as assessed by immunohistochemical detection of human beta(2)-microglobulin, were observed in the mouse tissue surrounding the transplanted human fragment. [less ▲] Detailed reference viewed: 16 (7 ULg)Transplantation of hepatocytes for prevention of intracranial hypertension in pigs with ischemic liver failure; ; et al in Cell Transplantation (1998), 7 Detailed reference viewed: 12 (4 ULg) |
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