2006
DOI: 10.1053/j.gastro.2006.01.008
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Sustained Engraftment and Tissue Enzyme Activity After Liver Cell Transplantation for Argininosuccinate Lyase Deficiency

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Cited by 193 publications
(139 citation statements)
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“…Hepatocyte isolation was performed by using two‐step collagenase perfusion under good manufacturing practice‐like conditions 22, 23, 24, 25. The isolated hepatocytes were stored in liquid nitrogen until use.…”
Section: Methodsmentioning
confidence: 99%
“…Hepatocyte isolation was performed by using two‐step collagenase perfusion under good manufacturing practice‐like conditions 22, 23, 24, 25. The isolated hepatocytes were stored in liquid nitrogen until use.…”
Section: Methodsmentioning
confidence: 99%
“…Also, a temporary, but impressive, reduction in the requirement for exogenous recombinant factor VII was achieved by transplantation of adult hepatocytes to two children with inherited severe factor VII deficiency, although orthotopic liver transplants were required by both recipients after 6 months [46]. A better success was seen in a 3 year-old patient with a urea cycle disorder, arginosuccinate lyase deficiency, where a heroic course of 11 hepatocyte transplants achieved a peak of 19% donor hepatocytes at 8 months [47].…”
Section: Cell Therapies Using Hepatocytesmentioning
confidence: 99%
“…Recently, hepatocyte transplantation has been reported to improve metabolic abnormalities present in the animal models of Wilson's disease, Crigler-Nijjar syndrome type I, and hereditary tyrosinaemia type 1 and lowered serum cholesterol levels in a rabbit model of homozygous familial hyper-cholesterolemia (Overturf et al, 1996;Gunsalus et al, 1997;Park et al, 2006). Furthermore, promising results have been also demonstrated from hepatocyte transplantation in human diseases including glycogen storage disease type 1a, Crigler-Najjar syndrome type I, argininosuccinate lyase deficiency, and inherited factor VII deficiency (Fox et al, 1998;Muraca et al, 2002;Dhawan et al, 2004;Stephenne et al, 2006). However, only less than 100 hepatocyte transplantation cases have been reported in humans up to date.…”
Section: Introductionmentioning
confidence: 99%