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  • Title: [Intrasplenic transplantation of hepatocytes in spf-ash mice with congenital ornithine transcarbamylase deficiency].
    Author: Michel JL, Rabier D, Rambaud C, Kamoun P, Brousse N, Vassault A, Pla M, Calise D, Revillon Y.
    Journal: Chirurgie; ; 119(10):666-71. PubMed ID: 7729184.
    Abstract:
    The only curative treatment for enzymatic deficiency such as Ornithine transcarbamylase (OTC) deficiency is liver transplantation. We have studied hepatocyte transplantation as alternative therapy in spfash mouse, the animal mode of OTC deficiency. Hepatocytes were obtained from C57BL/6J strain mice (normal OTC activity). About 2 x 10(6) hepatocytes in 0.1 ml were injected in the spleen of recipient mouse. We then studied: ammonemia, urinary orotate, spleen OTC activity, liver OTC activity, histological presence of hepatocytes in the spleen, immunohistochemical OTC staining in liver and spleen hepatocytes. In syngeneic hepatocyte transplantation (C57 > C57) functional transplanted hepatocytes were found in the spleen over 5 months after transplantation. In allogeneic transplantation (C57 > spfash) without immunosuppression, hepatocytes were rejected in 3 days. With cyclosporine immunosuppression, hepatocyte survived 12 days and showed OTC activity. A few transplanted hepatocytes with OTC activity migrated from spleen of liver. Metabolic disorder were not corrected. This technically easy method (compared to whole liver transplantation) demonstrated 2 problems: reject in allogeneic transplantation and the small amount of injected enzyme.
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