Application | Comment | Organism |
---|---|---|
diagnostics | sulfatases are potential therapeutic biopharmaceuticals | Homo sapiens |
Cloned (Comment) | Organism |
---|---|
the recombinant fusion enzyme protein is triaged to the lysosomal compartment of MPS-II fibroblasts, expression in CHO cells as IgG-sulfatase fusion protein with the enzyme fused to a genetically engineered monoclonal antibody against the human insulin receptor, i.e. HIRMAb. The cDNA encoding the human IDS cDNA, minus the sequence encoding the signal peptide, is fused to the carboxyl terminus of the CH3 region of the heavy chain of the chimeric HIRMAb | Homo sapiens |
Protein Variants | Comment | Organism |
---|---|---|
additional information | to deliver the enzyme across the human blood-brain barrier, the sulfatase is re-engineered as an IgG-sulfatase fusion protein with a genetically engineered monoclonal antibody against the human insulin receptor. The human insulin receptor monoclonal antibody HIRMAb part of the HIRMAb-IDS fusion protein acts as a molecular Trojan horse to ferry the fused IDS across the blood-brain barrier, overview. The HIRMAb-IDS fusion protein is tritiated and injected intravenously into the adult Rhesus monkey at a low dose of 0.1 mg/kg. The IDS enzyme activity in plasma is elevated 10fold above the endogenous level, and therapeutic plasma concentrations are generated in vivo. The uptake of the HIRMAb-IDS fusion protein in the brain is sufficiently high to produce therapeutic concentrations of IDS in the brain following IV administration of the fusion protein | Homo sapiens |
Organism | UniProt | Comment | Textmining |
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Homo sapiens | - |
- |
- |
Synonyms | Comment | Organism |
---|---|---|
IDS | - |
Homo sapiens |
General Information | Comment | Organism |
---|---|---|
malfunction | mucopolysaccharidosis, MPS, type II is caused by mutations in the lysosomal enzyme, iduronate-2-sulfatase. MPS-II affects the brain and enzyme replacement therapy is ineffective for the brain, because the enzyme does not cross the blood-brain barrier | Homo sapiens |