Long-term expressed human alpha-galactosidase A in tissues of HalphaG transgenic mice.

BACKGROUND

Human alpha-galactosidase A (halphaG) is an essential lysosomal enzyme in catalyzing the hydrolysis of ceramide trihexoside in humans. Effects have been directed to develop effective gene and replacement therapies for the deficiency of halphaG, Fabry disease. In recent years, halphaG transgenic mice (TGM) have been established, and the expression of halphaG in their general organs has been reported. However, detailed distribution of the cells expressing halphaG have not yet been defined.

METHODS

The distribution of halphaG in organs of the halphaG-TGM was studied by means of immunohistochemistry and enzyme assay.

RESULTS

Immunohistochemical analysis revealed a systematic halphaG expression in the TGM, including endothelial cells of the bone marrow, liver, spleen, pancreas, lungs, uriniferous tubules in the kidneys, and choroids plexus in the brain. Enzyme assay demonstrated a persistent expression of halphaG in the TGM during 14-20 months after birth.

CONCLUSION

A long-term expression of halphaG in organs may indicate halphaG-TGM as a useful tool in the research of gene and replacement therapies for Fabry disease.