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Originally published In Press as doi:10.1074/jbc.M107778200 on September 18, 2001

J. Biol. Chem., Vol. 276, Issue 46, 43160-43165, November 16, 2001
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Biodistribution, Kinetics, and Efficacy of Highly Phosphorylated and Non-phosphorylated beta -Glucuronidase in the Murine Model of Mucopolysaccharidosis VII*

Mark S. SandsDagger §, Carole A. Vogler||, Kevin K. Ohlemiller**, Marie S. RobertsDagger , Jeffrey H. GrubbDagger Dagger , Beth Levy||, and William S. SlyDagger Dagger

From the Departments of Dagger  Internal Medicine and § Genetics, and the ** Research Department, Central Institute for the Deaf, Washington University School of Medicine, St. Louis, Missouri 63110 and the || Department of Pathology and the Dagger Dagger  Edward A. Doisy Department of Biochemistry and Molecular Biology, St. Louis University School of Medicine, St. Louis, Missouri 63104

Enzyme replacement therapy (ERT) has been shown to be effective at reducing the accumulation of undegraded substrates in lysosomal storage diseases. Most ERT studies have been performed with recombinant proteins that are mixtures of phosphorylated and non-phosphorylated enzyme. Because different cell types use different receptors to take up phosphorylated or non-phosphorylated enzyme, it is difficult to determine which form of enzyme contributed to the clinical response. Here we compare the uptake, distribution, and efficacy of highly phosphorylated and non-phosphorylated beta -glucuronidase (GUSB) in the MPS VII mouse. Highly phosphorylated murine GUSB was efficiently taken up by a wide range of tissues. In contrast, non-phosphorylated murine GUSB was taken up primarily by tissues of the reticuloendothelial (RE) system. Although the tissue distribution was different, the half-lives of both enzymes in any particular tissue were similar. Both preparations of enzyme were capable of preventing the accumulation of lysosomal storage in cell types they targeted. An important difference in clinical efficacy emerged in that phosphorylated GUSB was more efficient than non-phosphorylated enzyme at preventing the hearing loss associated with this disease. These data suggest that both forms of enzyme contribute to the clinical responses of ERT in MPS VII mice but that enzyme preparations containing phosphorylated GUSB are more broadly effective than non-phosphorylated enzyme.

* This work was supported by National Institutes of Health Grants RO1 HD35671 (to M. S. S.) and RO1 GM34182 (to W. S. S.) and The Edward Mallinckrodt Jr. Foundation (to K. K. O.).The costs of publication of this article were defrayed in part by the payment of page charges. The article must therefore be hereby marked "advertisement" in accordance with 18 U.S.C. Section 1734 solely to indicate this fact.

To whom correspondence should be addressed: Dept. of Internal Medicine, Washington University School of Medicine, Box 8007, 660 South Euclid Ave., St. Louis, MO 63110. Tel.: 314-362-5494; Fax: 314-362-9333; E-mail: msands@imgate.wustl.edu.

Copyright © 2001 by The American Society for Biochemistry and Molecular Biology, Inc.
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