Abstract: The invention relates to a method of treating mucopolysaccharidoses using enzyme replacement therapy with chemically modified lysosomal enzymes. More specifically the method relates to administering chemically modified lysosomal enzymes intraperitoneal injection. In addition, the invention relates to treating type VII mucopolysaccharidoses or mucopolysaccharidoses type VII related bone lesions with a chemical modified ?-glucuronidase, wherein the modified ?-glucuronidase may be administered 5 weeks after birth, and or may be administered intraperitoneally.

Abstract: The present invention provides compositions and methods for use in enzyme replacement therapy. The inventors disclose a method of producing membrane bound enzymes in an active soluble form by eliminating the glycosylphosphatidylinositol (GPI) membrane anchor. In particular the inventors disclose a soluble active form of the membrane bound enzyme TNSALP which they produced by deleting the GPI anchor single peptide sequence. They have further shown that this composition is useful for treatment of hypophosphatasia. The inventors also disclose oligo acid amino acid variants thereof which specifically target bone tissue.

Abstract: The present invention provides compositions and methods for use in enzyme replacement therapy. The inventors disclose a method of producing membrane bound enzymes in an active soluble form by eliminating the glycosylphosphatidylinositol (GPI) membrane anchor. In particular the inventors disclose a soluble active form of the membrane bound enzyme TNSALP which they produced by deleting the GPI anchor single peptide sequence. They have further shown that this composition is useful for treatment of hypophosphatasia. The inventors also disclose oligo acid amino acid variants thereof which specifically target bone tissue.

Abstract: The invention is directed to chimeral fusion proteins having an IgG1 antibody Fc portion and a lysosomal storage enzyme, particularly a Fc-GUS fusion protein useful in treating Sly's disease in an embryo or fetus. The invention is also directed to methods of treating in born errors of metabolism, particularly Sly's disease, in a fetus by delivering to a pregnant mother a Fc-MPS emzyme fusion protein.

Abstract: The present invention provides a polypeptide therapeutic agent, useful in enzyme replacement therapy, with increased therapeutic benefits for the central nervous system. The invention provides a method of enhancing the effect of a polypeptide or protein on the central nervous system by the attachment of a short acidic amino acid sequence. Specifically the inventors disclose the attachment of a 4-15 acidic amino acid sequence to human ?-glucuronidase by construction of a fusion protein. This molecule is useful in the treatment of type VII mucopolysaccharidosis when administered to a patient.

Abstract: The present invention provides compositions and methods for use in enzyme replacement therapy. The inventors disclose a method of producing membrane bound enzymes in an active soluble form by eliminating the glycosylphosphatidylinositol (GPI) membrane anchor. In particular the inventors disclose a soluble active form of the membrane bound enzyme TNSALP which they produced by deleting the GPI anchor single peptide sequence. They have further shown that this composition is useful for treatment of hypophosphatasia. The inventors also disclose oligo acid amino acid variants thereof which specifically target bone tissue.

Abstract: An ophthalmic fluid containing at least one triglyceride, diglyceride, or monoglyceride which is chemically similar or compatible with compounds found naturally in the lipid layer of the tear film of an ocular substrate. Preferably the glycerides are derived from a fatty acid containing at least one unsaturated bond in a cis-configuration in the fatty acid residue. The ophthalmic fluid is arranged in use to provide a protective film across a tear film of an ocular substrate. Furthermore, the ophthalmic fluid is also arranged in use to provide a protective film intermediate a lipid layer of the tear film of the ocular substrate and a contact lens.

Abstract: Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.

Abstract: An ophthalmic fluid containing at least one triglyceride, diglyceride, or monoglyceride which is chemically similar or compatible with compounds found naturally in the lipid layer of the tear film of an ocular substrate. Preferably the glycerides are derived from a fatty acid containing at least one unsaturated bond in a cis-configuration in the fatty acid residue. The ophthalmic fluid is arranged in use to provide a protective film across a tear film of an ocular substrate. Furthermore, the ophthalmic fluid is also arranged in use to provide a protective film intermediate a lipid layer of the tear film of the ocular substrate and a contact lens.