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Updated: Feb 1, 2026
Orthotopic Liver Transplantation in Rats
Published on: July 1, 2012
Liver transplantation: New treatment for mucopolysaccharidosis type VI in rats
Sumika Toyama1,2, Ohsuke Migita3, Masayuki Fujino1,4
1Division of Transplantation Immunology, National Center for Child Health and Development, Tokyo, Japan.
Background:
Mucopolysaccharidosis (MPS) VI is a rare, autosomal recessive congenital metabolic disorder caused by deficient activity of the lysosomal metabolic enzyme, N-acetylgalactosamine 4-sulfatase. Enzyme replacement therapy (ERT) is the current treatment for MPS VI, although it involves limited compliance to the therapy and high cost. The aim of this study was to develop a new method of treatment by conducting an orthotopic liver transplantation (LTx) using an animal model of human MPS VI, and to evaluate and examine its effectiveness for treating MPS VI.
Methods:
LTx was carried out from normal unaffected to affected MPS VI rats (MPR), which were then killed after LTx, and tissues from the heart, spleen, and knee joint, as well as serum, collected for biological and morphologic evaluation.
Results:
Liver-transplanted (LTx) MPR had the same level of N-acetylgalactosamine 4-sulfatase activity in the liver and lungs as normal unaffected MPR, and the urinary secretion of mucopolysaccharides/glycosaminoglycan (GAG) in LTx MPR was significantly decreased. Furthermore, on histopathology, the spleens of LTx MPR showed elimination of vacuole cells. In the knee joints, growth plates became thinner, and on radiography the facial and cranial bones of LTx MPR were morphologically normal.
Conclusions:
LTx from normal to affected MPR was effective for symptoms of MPS and accumulation of GAG, suggesting that LTx could be a promising alternative approach for MPS VI.
Insights
Liver transplantation (LTx) effectively treated Mucopolysaccharidosis (MPS) VI in rats by restoring enzyme activity and reducing GAG accumulation. This suggests LTx is a promising alternative therapy for MPS VI patients.
Area of Science:
- Biochemistry
- Genetics
- Transplantation
Background:
- Mucopolysaccharidosis (MPS) VI is a rare metabolic disorder due to N-acetylgalactosamine 4-sulfatase deficiency.
- Current enzyme replacement therapy (ERT) for MPS VI has limitations in compliance and cost.
Purpose of the Study:
- To develop and evaluate orthotopic liver transplantation (LTx) as a novel treatment for MPS VI.
- To assess the effectiveness of LTx in an animal model of human MPS VI.
Main Methods:
- Orthotopic liver transplantation (LTx) was performed from normal rats to MPS VI rats (MPR).
- Biological and morphological evaluations were conducted on tissues (heart, spleen, knee joint) and serum post-LTx.
Main Results:
- LTx restored N-acetylgalactosamine 4-sulfatase activity in the liver and lungs of MPR.
- Urinary mucopolysaccharide/glycosaminoglycan (GAG) excretion significantly decreased in LTx MPR.
- Histopathology showed spleen vacuole cell elimination, normalized growth plates, and normal facial/cranial bones in LTx MPR.
Conclusions:
- LTx from normal to affected MPR effectively ameliorated MPS VI symptoms and GAG accumulation.
- Liver transplantation (LTx) shows promise as an alternative therapeutic strategy for Mucopolysaccharidosis (MPS) VI.
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