ArticleProceedings of the National Academy of Sciences of the United States of America2008
Infused Fc-tagged beta-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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Who cites it
19 citing papers in PubMed, 37 citations in OpenAlex.
- Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system.Molecular genetics and metabolism reports · 2022Article
- Relevance of the Materno-Fetal Interface for the Induction of Antigen-Specific Immune Tolerance.Frontiers in immunology · 2020Review
- Tolerating Factor VIII: Recent Progress.Frontiers in immunology · 2019Review
- The Neonatal Fc Receptor (FcRn): A Misnomer?Frontiers in immunology · 2019Review
- Review
- Mutations of Recombinant Aquaporin-4 Antibody in the Fc Domain Can Impair Complement-Dependent Cellular Cytotoxicity and Transplacental Transport.Frontiers in immunology · 2018Article
- Pathogenesis and treatment of spine disease in the mucopolysaccharidoses.Molecular genetics and metabolism · 2016Review
- Unraveling the Interaction between FcRn and Albumin: Opportunities for Design of Albumin-Based Therapeutics.Frontiers in immunology · 2014Review
- Regulation of immune responses by the neonatal fc receptor and its therapeutic implications.Frontiers in immunology · 2014Review
- Assessment of bone dysplasia by micro-CT and glycosaminoglycan levels in mouse models for mucopolysaccharidosis type I, IIIA, IVA, and VII.Journal of inherited metabolic disease · 2013Article
- Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model.Molecular genetics and metabolism · 2012Article
- Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs.Blood · 2012Article
- Neonatal Fc receptor: from immunity to therapeutics.Journal of clinical immunology · 2010Review
- Therapeutic approaches for neuronopathic lysosomal storage disorders.Journal of inherited metabolic disease · 2010Review
- Guanidinylated neomycin mediates heparan sulfate-dependent transport of active enzymes to lysosomes.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Chapter 4: Multitasking by exploitation of intracellular transport functions the many faces of FcRn.Advances in immunology · 2009Review
- Aptamer-based endocytosis of a lysosomal enzyme.Proceedings of the National Academy of Sciences of the United States of America · 2008Article
- New strategies for enzyme replacement therapy for lysosomal storage diseases.Rejuvenation researchReview
- Review
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Glycosaminoglycan storage begins in prenatal life in patients with mucopolysaccharidosis (MPS). In fact, prenatal hydrops is a common manifestation of MPS VII because of beta-glucuronidase (GUS) deficiency. One way to address prenatal storage might be to deliver the missing enzyme across the placenta into the fetal circulation. Maternal IgG is transported across the placenta by the neonatal Fc receptor (FcRn), which recognizes the Fc domain of IgG and mediates transcytosis from maternal to fetal circulation. We hypothesized that we could exploit this process to deliver corrective enzyme to the fetus. To test this hypothesis, the C-terminal fusion protein, GUS-Fc, was compared with native, untagged, recombinant GUS for clearance from the maternal circulation, delivery to the fetus, and reduction of lysosomal storage in offspring of MPS VII mice. We observed that GUS-Fc, infused into pregnant mothers on embryonic days 17 and 18, was transported across the placenta. Similarly infused untagged GUS was not delivered to the fetus. GUS-Fc plasma enzyme activity in newborn MPS VII mice was 1,000 times that seen after administration of untagged GUS and approximately 100 times that of untreated WT newborns. Reduced lysosomal storage in heart valves, liver, and spleen provided evidence that in utero enzyme replacement therapy with GUS-Fc targeted sites of storage in the MPS VII fetus. We hypothesize that this noninvasive approach could deliver the missing lysosomal enzyme to a fetus with any lysosomal storage disease. It might also provide a method for inducing immune tolerance to the missing enzyme or another foreign protein.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.