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Blood, ISSN 0006-4971, 10/2015, Volume 126, Issue 15, pp. 1777 - 1784
Journal Article
Human Gene Therapy, ISSN 1043-0342, 08/2017, Volume 28, Issue 8, pp. 626 - 638
Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked recessive lysosomal disorder caused by defective iduronate-2-sulfatase (IDS),... 
Research Articles | Hunter syndrome | gene therapy | neurocognitive function | mucopolysaccharidosis type II | AAV | MEDICINE, RESEARCH & EXPERIMENTAL | HURLER-SYNDROME | STORAGE DISEASE | BONE-MARROW-TRANSPLANTATION | CNS | DELIVERY | MURINE MODEL | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | MOUSE MODEL | GENETICS & HEREDITY | BLOOD-BRAIN | ENZYME REPLACEMENT THERAPY | EXPRESSION | Dependovirus - genetics | Genetic Therapy | Central Nervous System - metabolism | Genetic Vectors - administration & dosage | Iduronate Sulfatase - metabolism | Humans | Male | Cognition | Tissue Distribution | Time Factors | Mucopolysaccharidosis II - therapy | Mucopolysaccharidosis II - genetics | Female | Gene Order | Disease Models, Animal | Gene Transfer Techniques | Gene Expression | Iduronate Sulfatase - blood | Transduction, Genetic | Glycosaminoglycans - metabolism | Genetic Vectors - genetics | Neuropsychological Tests | Mucopolysaccharidosis II - blood | Pilot Projects | Animals | Iduronate Sulfatase - genetics | Mice | Enzyme Activation | Mucopolysaccharidosis II - psychology | Heparan sulfate | Brain | Animal models | Glycosaminoglycans | Gene transfer | Central nervous system | Organs | Disorders | Viruses | Nervous system | Mucopolysaccharidosis | Injection | Sulfates | Tissues | Hereditary diseases | Prevention | Gag protein | Sulfate | Circulation
Journal Article
Biochemical Journal, ISSN 0264-6021, 03/2014, Volume 458, Issue 2, pp. 281 - 289
Journal Article
Journal Article
Journal Article
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 2011, Volume 6, Issue 1, pp. 72 - 72
Journal Article
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 09/2018, Volume 125, Issue 1-2, pp. 53 - 58
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2018, Volume 26, Issue 5, pp. 1366 - 1374
Mucopolysaccharidosis II (MPS II) is an X-linked recessive lysosomal storage disease caused by mutations in the iduronate-2-sulfatase ( ) gene. Since IDS... 
blood-brain barrier | glycosaminoglycans | enzyme replacement therapy | iduronate-2-sulfatase | mucopolysaccharidosis II | lysosomal storage disease | transferrin receptor | HUMAN INSULIN-RECEPTOR | MEDICINE, RESEARCH & EXPERIMENTAL | 6-PHOSPHATE RECEPTOR | PRIMATE | RHESUS-MONKEYS | MONOCLONAL-ANTIBODY | REDUCES GLYCOSOAMINOGLYCANS | HUNTER-SYNDROME | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | DISEASE | GENETICS & HEREDITY | IDURONIDASE | ENZYME REPLACEMENT THERAPY | Cell Line | Tissue Distribution - drug effects | Antibodies, Monoclonal - pharmacology | Humans | Antibodies, Monoclonal - pharmacokinetics | Blood-Brain Barrier - drug effects | Receptor, IGF Type 2 - metabolism | Recombinant Fusion Proteins | Blood-Brain Barrier - metabolism | Mice, Knockout | Brain - drug effects | Brain - metabolism | Receptor, IGF Type 2 - genetics | Animals | Mucopolysaccharidosis II - metabolism | Antibodies, Monoclonal - administration & dosage | Mucopolysaccharidosis II - drug therapy | Fibroblasts - drug effects | Mucopolysaccharidosis II - genetics | Mice | Receptors, Transferrin - antagonists & inhibitors | Disease Models, Animal | Fibroblasts - metabolism | Heparan sulfate | Brain | Enzymes | Transferrin | Animal models | Intravenous administration | Glycosaminoglycans | Statistical analysis | Lysosomal storage diseases | Mucopolysaccharidosis | Monkeys & apes | Experiments | Neurological diseases | Proteins | Blood-brain barrier | Fibroblasts | Fusion protein | Drug dosages | Binding sites | Original
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