Infused Fc-tagged β-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice

Proceedings of the National Academy of Sciences of the United States of America

Volumn 105, Issue 24, 2008, Pages 8375-8380

  Grubb, Jeffrey H ^a   Vogler, Carole ^a   Tan, Yun ^a   Shah, Gul N ^a   MacRae, Amy F ^a   Sly, William S ^a  

^a SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Enzyme replacement therapy; Lysosomal storage disease; Receptor mediated endocytosis; Transplacental delivery

Indexed keywords

BETA GLUCURONIDASE FC FUSION PROTEIN; GLYCOSAMINOGLYCAN; HYBRID PROTEIN; IMMUNOGLOBULIN G; RECOMBINANT PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CARBOXY TERMINAL SEQUENCE; CLEARANCE; CONTINUOUS INFUSION; CONTROLLED STUDY; EMBRYO; ENZYME ACTIVITY; ENZYME REPLACEMENT; FEMALE; FETUS CIRCULATION; FETUS HYDROPS; IMMUNOLOGICAL TOLERANCE; LYSOSOME STORAGE DISEASE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NEWBORN; NONHUMAN; NUCLEOTIDE SEQUENCE; PLACENTAL TRANSFER; PRENATAL PERIOD; PRIORITY JOURNAL; PROGENY; PROTEIN DOMAIN; TRANSCYTOSIS;

ANIMALS; FEMALE; GLUCURONIDASE; GLYCOSAMINOGLYCANS; INFUSIONS, PARENTERAL; LYSOSOMES; MICE; MUCOPOLYSACCHARIDOSIS VII; PLACENTA; PREGNANCY; RECEPTORS, FC; RECOMBINANT FUSION PROTEINS; TISSUE DISTRIBUTION; UTERUS;

MUS;

EID: 46149113010     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.0803715105     Document Type: Article

References (25)

1. Neufeld EF, Muenzer J (2001) The Mucopolysaccharidoses. The Metabolic and Molecular Bases of Inherited Disease, eds Scriver CR, Beaudet AL, Sly WS, Valle D (McGraw-Hill, New York), pp 3421-3451.
2. Brady RO (2006) Enzyme replacement for lysosomal diseases. Annu Rev Med 57:283-296.
3. Sands MS, et al. (2001) Biodistribution, kinetics, and efficacy of highly phosphorylated and nonphosphorylated β-glucuronidase in the murine model of mucopolysaccharidosis VII. J Biol Chem 276:43160-43165.
4. Sly WS, et al. (2006) Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors. Proc Natl Acad Sci USA 103:15172-15177.
5. Cheng Y, Verp MS, Knutel T, Hibbard JU (2003) Mucopolysaccharidosis type VII as a cause of recurrent nonimmune hydrops fetalis. J Perinat Med 31:535-537.
6. Roopenian DC, Akilesh S (2007) FcRn: The neonatal Fc receptor comes of age. Nat Rev Immunol 7:715-725.
7. Grubb JH, et al. (2008) Chemically modified β-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII. Proc Natl Acad Sci USA 105:2616-2621.
8. LeBowitz JH, et al. (2004) Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice. Proc Natl Acad Sci USA 101:3083-3088.
9. Orii KO, et al. (2005) Defining the pathway for Tat-mediated delivery of β-glucuronidase in cultured cells and MPS VII mice. Mol Ther 12:345-352.
10. Lee SJ, et al. (2002) Mannose receptor-mediated regulation of serum glycoprotein homeostasis. Science 295:1898-1901.
11. Roopenian DC, et al. (2003) The MHC class I-like IgG receptor controls perinatal IgG transport, IgG homeostasis, and fate of IgG-Fc-coupled drugs. J Immunol 170:3528-3533.
12. Koller BH, Smithies O (1989) Inactivating the β2-microglobulin locus in mouse embryonic stem cells by homologous recombination. Proc Natl Acad Sci USA 86:8932-8935.
13. Bitonti AJ, et al. (2004) Pulmonary delivery of an erythropoietin Fc fusion protein in nonhuman primates through an immunoglobulin transport pathway. Proc Natl Acad Sci USA 101:9763-9768.
14. Israel EJ, Patel VK, Taylor SF, Marshak-Rothstein A, Simister NE (1995) Requirement for a β2-microglobulin-associated Fc receptor for acquisition of maternal IgG by fetal and neonatal mice. J Immunol 154:6246-6251.
15. Van de PP (2003) Transfer of antibody via mother's milk. Vaccine 21:3374-3376.
16. Achord DT, Brot FE, Bell CE, Sly WS (1978) Human β-glucuronidase: In vivo clearance and in vitro uptake by a glycoprotein recognition system on reticuloendothelial cells. Cell 15:269-278.
17. Parnell SC, et al. (2002) Polycystin-1 activation of c-Jun N-terminal kinase and AP-1 is mediated by heterotrimeric G proteins. J Biol Chem 277:19566-19572.
18. Niwa H, Yamamura K, Miyazaki J (1991) Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108:193-199.
19. Ulmasov B, et al. (2000) Purification and kinetic analysis of recombinant CA XII, a membrane carbonic anhydrase overexpressed in certain cancers. Proc Natl Acad Sci USA 97:14212-14217.
20. Glaser JH, Sly WS (1973) β-Glucuronidase deficiency mucopolysaccharidosis: Methods for enzymatic diagnosis. J Lab Clin Med 82:969-977.
21. Lowry OH, Rosebrough NJ, Farr AL, Randall RJ (1951) Protein measurement with the folin phenol reagent. J Biol Chem 193:265-275.
22. Islam MR, Grubb JH, Sly WS (1993) C-terminal processing of human β-glucuronidase: The propeptide is required for full expression of catalytic activity, intracellular retention, and proper phosphorylation. J Biol Chem 268:22627-22633.
23. Laemmli UK (1970) Cleavage of structural proteins during the assembly of the head of bacteriophage T4. Nature 227:680-685.
24. Kaplan A, Achord DT, Sly WS (1977) Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts. Proc Natl Acad Sci USA 74:2026-2030.
25. Diment S, Leech MS, Stahl PD (1987) Generation of macrophage variants with 5-azacytidine: Selection for mannose receptor expression. J Leukocyte Biol 42:485-490.