Correction of β-glucuronidase deficiency in mice by intrasplenic transplantation of isolated hepatocytes

Correction of enzyme activity was successfully accomplished in β-glucuronidase deficient C3H/HeJ mice after intrasplenic transplantation of isolated hepatocytes obtained from normal Balb/C donors mice. Enzyme activity increased from a mean of 1.1 ± 0.2 nmol/h/ml on day 0 to 15.8 ± 3.3 nmol/h/ml on day 55 post-transplantation. Examination of enzyme activity in liver tissue by repeated liver biopsies revealed an increase in β-glucuronidase activity from a mean of 1.85 ± 0.6 nmol/h/mg protein on day 0 up to 42.6 ± 7.3 nmol/h/mg protein on day 35 post-transplantation. β-Galactosidase and β-N-acetyl-glucosaminidase activities were not significantly changed throughout the study. Intrasplenic transplantation of hepatocytes isolated from β-glucuronidase-deficient mice did not induce a significant change in three examined hydrolases. These results suggest that transplantation of isolated hepatocytes may be used as an effective tool for therapy of lysosomal enzyme deficiency in mice.