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1. Full Text In vivo genome editing of the albumin locus as a platform for protein replacement therapy
by Sharma, Rajiv and Anguela, Xavier M and Doyon, Yannick and Wechsler, Thomas and DeKelver, Russell C and Sproul, Scott and Paschon, David E and Miller, Jeffrey C and Davidson, Robert J and Shivak, David and Zhou, Shangzhen and Rieders, Julianne and Gregory, Philip D and Holmes, Michael C and Rebar, Edward J and High, Katherine A
Blood, ISSN 0006-4971, 10/2015, Volume 126, Issue 15, pp. 1777 - 1784
Site-specific genome editing provides a promising approach for achieving long-term, stable therapeutic gene expression. Genome editing has been successfully... 
SIGNAL | SEROTYPE | ADENOASSOCIATED VIRUS | GENE-TRANSFER | HEMATOLOGY | ZINC-FINGER NUCLEASES | EXPRESSION | T-CELLS | Hemophilia B - therapy | Dependovirus - genetics | Genetic Therapy | Transgenes - physiology | Genetic Vectors - administration & dosage | Fabry Disease - genetics | RNA Editing | Humans | Lysosomes - enzymology | Gaucher Disease - therapy | Promoter Regions, Genetic - genetics | Mucopolysaccharidosis I - therapy | Mucopolysaccharidosis II - therapy | Mucopolysaccharidosis II - genetics | Real-Time Polymerase Chain Reaction | Enzyme Replacement Therapy | Zinc Fingers | Albumins - metabolism | Liver - metabolism | Mice, Inbred C57BL | RNA, Messenger - genetics | Factor VIII - genetics | Factor IX - genetics | Hemophilia B - genetics | Hemophilia A - therapy | Reverse Transcriptase Polymerase Chain Reaction | Animals | Gaucher Disease - genetics | Endonucleases | Mucopolysaccharidosis I - genetics | Hemophilia A - genetics | High-Throughput Nucleotide Sequencing | Mice | Fabry Disease - therapy | Genome | Albumins - genetics | Gene Therapy
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2. Full Text Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer
by Laoharawee, Kanut and Podetz-Pedersen, Kelly M and Nguyen, Tam T and Evenstar, Laura B and Kitto, Kelley F and Nan, Zhenhong and Fairbanks, Carolyn A and Low, Walter C and Kozarsky, Karen F and McIvor, R. Scott
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
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3. Full Text Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts
by Kan, Shih-Hsin and Troitskaya, Larisa A and Sinow, Carolyn S and Haitz, Karyn and Todd, Amanda K and Di Stefano, Ariana and Le, Steven Q and Dickson, Patricia I and Tippin, Brigette L
Biochemical Journal, ISSN 0264-6021, 03/2014, Volume 458, Issue 2, pp. 281 - 289
Enzyme replacement therapy for MPS IIIB (mucopolysaccharidosis type IIIB; also known as Sanfilippo B syndrome) has been hindered by inadequate mannose 6... 
Mucopolysaccharidosis | Lysosomal storage disease | Enzyme replacement therapy | Sanfilippo | Insulin-like growth factor (IGF) | enzyme replacement therapy | NEUROLOGICAL DISEASE | GENE DELIVERY | lysosomal storage disease | SANFILIPPO-SYNDROME | BIOCHEMISTRY & MOLECULAR BIOLOGY | mucopolysaccharidosis | SYNDROME TYPE-B | CEREBROSPINAL-FLUID | ENZYME-REPLACEMENT THERAPY | MURINE MODEL | insulin-like growth factor (IGF) | HAMSTER OVARY CELLS | MOUSE MODEL | CENTRAL-NERVOUS-SYSTEM | Fibroblasts - enzymology | Protein Binding - genetics | Cricetulus | Humans | Lysosomes - genetics | Lysosomes - enzymology | Acetylglucosaminidase - genetics | Recombinant Fusion Proteins - metabolism | Insulin-Like Growth Factor II - genetics | Lysosomes - metabolism | Acetylglucosaminidase - metabolism | Mucopolysaccharidosis III - metabolism | Endocytosis - genetics | Acetylglucosaminidase - biosynthesis | CHO Cells | Fibroblasts - metabolism | Cricetinae | Mucopolysaccharidosis III - genetics | Up-Regulation - genetics | Binding Sites - genetics | Fibroblasts - pathology | Mucopolysaccharidosis III - enzymology | Animals | Cell Line, Tumor | Recombinant Fusion Proteins - genetics | Amino Acid Motifs - genetics | insulin-like growth factor
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4. Full Text Clinical, biochemical and molecular characteristics of Filipino patients with mucopolysaccharidosis type II - Hunter syndrome
by Chiong, Mary Anne D and Canson, Daffodil M and Abacan, Mary Ann R and Baluyot, Melissa Mae P and Cordero, Cynthia P and Silao, Catherine Lynn T
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 01/2017, Volume 12, Issue 1, p. 7
Background: Mucopolysaccharidosis type II, an X-linked recessive disorder is the most common lysosomal storage disease detected among Filipinos. This is a case... 
Iduronate-2-sulfatase gene | Mucopolysaccharidosis type II | Hunter syndrome | Glycosaminoglycans | Lysosomal storage disease | MUTATION ANALYSIS | MEDICINE, RESEARCH & EXPERIMENTAL | DIAGNOSIS | IDENTIFICATION | MILD | DISEASE | GENETICS & HEREDITY | IDS GENE | JAPANESE PATIENTS | Glycosaminoglycans - blood | Iduronate Sulfatase - metabolism | Glycosaminoglycans - metabolism | Humans | Philippines | Lysosomal Storage Diseases - genetics | Exons - genetics | Male | Mutation, Missense - genetics | Mucopolysaccharidosis II - blood | Mucopolysaccharidosis II - metabolism | Adolescent | Iduronate Sulfatase - genetics | Lysosomal Storage Diseases - metabolism | Mucopolysaccharidosis II - genetics | Female | Lysosomal Storage Diseases - blood | Mutation | Child | Codon, Nonsense - genetics | Frameshift Mutation - genetics | Case studies | Enzymes | Gene mutations | Mucopolysaccharidosis | Genetic aspects | Research | Diagnosis | Health aspects
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5. Full Text Molecular diagnosis of 65 families with mucopolysaccharidosis type II (Hunter syndrome) characterized by 16 novel mutations in the IDS gene: Genetic, pathological, and structural studies on iduronate-2-sulfatase
by Kosuga, Motomichi and Mashima, Ryuichi and Hirakiyama, Asami and Fuji, Naoko and Kumagai, Tadayuki and Seo, Joo-Hyun and Nikaido, Mari and Saito, Seiji and Ohno, Kazuki and Sakuraba, Hitoshi and Okuyama, Torayuki
Molecular Genetics and Metabolism, ISSN 1096-7192, 07/2016, Volume 118, Issue 3, pp. 190 - 197
Mucopolysaccharidosis type II (MPS II: also called as Hunter syndrome) is an X-linked recessive lysosomal storage disorder characterized by the accumulation of... 
Genotypephenotype correlation | Hunter syndrome | Iduronate-2-sulfatase | MPS type II | Homology modeling | MEDICINE, RESEARCH & EXPERIMENTAL | MPS II | TASSER | AMINO-ACID SUBSTITUTIONS | READ-THROUGH | INVERSION | PREDICTION | DISEASE | ENDOCRINOLOGY & METABOLISM | GENETICS & HEREDITY | JAPANESE PATIENTS | ENZYME REPLACEMENT THERAPY | Glycoproteins - genetics | Genetic Predisposition to Disease | Protein Structure, Secondary | Humans | Japan | Asian Continental Ancestry Group - genetics | Models, Molecular | Male | Mucopolysaccharidosis II - pathology | Pedigree | Mucopolysaccharidosis II - genetics | Female | Structural Homology, Protein | Mutation | Glycoproteins - chemistry | Glycosaminoglycans - urine | Genetic research | Enzymes | Mucopolysaccharidosis | Genetic aspects | Genes
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6. Full Text Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II
by Wakabayashi, Taichi and Shimada, Yohta and Akiyama, Kazumasa and Higuchi, Takashi and Fukuda, Takahiro and Kobayashi, Hiroshi and Eto, Yoshikatsu and Ida, Hiroyuki and Ohashi, Toya
Human Gene Therapy, ISSN 1043-0342, 06/2015, Volume 26, Issue 6, pp. 357 - 366
Mucopolysaccharidosis type II (MPS II) is a neuropathic lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS), which leads to the... 
Research Articles | MEDICINE, RESEARCH & EXPERIMENTAL | ACTIVATION | EFFICACY | BONE-MARROW-TRANSPLANTATION | ALPHA-GLUCOSIDASE | METACHROMATIC LEUKODYSTROPHY | OVEREXPRESSION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | MOUSE MODEL | GENETICS & HEREDITY | MICE | CNS DEFECTS | ENZYME REPLACEMENT THERAPY | Glycoproteins - genetics | Transduction, Genetic | Iduronate Sulfatase - metabolism | Glycosaminoglycans - metabolism | Mice, Inbred C57BL | Glycoproteins - metabolism | Male | Mice, Knockout | Brain - metabolism | Animals | Mucopolysaccharidosis II - therapy | Brain - pathology | Hematopoietic Stem Cells - physiology | Iduronate Sulfatase - genetics | Mucopolysaccharidosis II - genetics | Female | Lentivirus - genetics | Hematopoietic Stem Cell Transplantation - methods | Disease Models, Animal | Genetic Therapy - methods
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7. Full Text Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease
by Scarpa, Maurizio and Almássy, Zsuzsanna and Beck, Michael and Bodamer, Olaf and Bruce, Iain A and De Meirleir, Linda and Guffon, Nathalie and Guillén-Navarro, Encarna and Hensman, Pauline and Jones, Simon and Kamin, Wolfgang and Kampmann, Christoph and Lampe, Christina and Lavery, Christine A and Leão Teles, Elisa and Link, Bianca and Lund, Allan M and Malm, Gunilla and Pitz, Susanne and Rothera, Michael and Stewart, Catherine and Tylki-Szymaska, Anna and Van Der Ploeg, Ans and Walker, Robert and Zeman, Jiri and Wraith, James E and Hunter Syndrome Europena Expert Council
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 2011, Volume 6, Issue 1, pp. 72 - 72
Mucopolysaccharidosis type II (MPS II) is a rare, life-limiting, X-linked recessive disease characterised by deficiency of the lysosomal enzyme... 
MEDICINE, RESEARCH & EXPERIMENTAL | LARYNGEAL MASK AIRWAY | HURLER-SYNDROME | BONE-MARROW-TRANSPLANTATION | HUNTER OUTCOME SURVEY | GENETICS & HEREDITY | TERM-FOLLOW-UP | SLEEP-APNEA | CARPAL-TUNNEL-SYNDROME | LYSOSOMAL STORAGE DISEASES | ENZYME REPLACEMENT THERAPY | STEM-CELL TRANSPLANT | Rare Diseases - genetics | Enzyme Replacement Therapy | Humans | Rare Diseases - drug therapy | Rare Diseases - diagnosis | Male | Treatment Outcome | Clinical Trials as Topic | Mucopolysaccharidosis II - diagnosis | Mucopolysaccharidosis II - pathology | Rare Diseases - therapy | Adolescent | Mucopolysaccharidosis II - therapy | Mucopolysaccharidosis II - genetics | Adult | Female | Rare Diseases - pathology | Disease Management | Iduronate Sulfatase - therapeutic use | Enzymes | Care and treatment | Stem cells | Development and progression | Mucopolysaccharidosis | Transplantation | Diagnosis | Research | Health aspects | Health care | Pediatrics | Hospitals | Meetings | Colleges & universities | Otolaryngology | Clinical trials | Genetics | Administrative support | Respiratory system | Metabolic disorders | Manuscripts
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8. Full Text Identification and characterization of 20 novel pathogenic variants in 60 unrelated Indian patients with mucopolysaccharidoses type I and type II
by Uttarilli, A and Ranganath, P and Matta, D and Md Nurul Jain, J and Prasad, K and Babu, A.S and Girisha, K.M and Verma, I.C and Phadke, S.R and Mandal, K and Puri, R.D and Aggarwal, S and Danda, S and Sankar, V.H and Kapoor, S and Bhat, M and Gowrishankar, K and Hasan, A.Q and Nair, M and Nampoothiri, S and Dalal, A
Clinical Genetics, ISSN 0009-9163, 12/2016, Volume 90, Issue 6, pp. 496 - 508
Mucopolysaccharidoses (MPS), a subgroup of lysosomal storage disorders, are caused due to deficiency of specific lysosomal enzyme involved in catabolism of... 
genotype–phenotype correlation | Hunter syndrome | Hurler syndrome | mucopolysaccharidoses | variant | mucopolysaccharidosis type II | mucopolysaccharidosis type I | GENE | SERVER | genotype-phenotype correlation | DISEASE | GENETICS & HEREDITY | MUTATIONS | Glycoproteins - genetics | Humans | Mucopolysaccharidosis I - physiopathology | Child, Preschool | Infant | Male | Structure-Activity Relationship | Mutation - genetics | India | Iduronidase - chemistry | Phenotype | Mucopolysaccharidosis II - physiopathology | Adolescent | Mucopolysaccharidosis II - genetics | Female | Mucopolysaccharidosis I - genetics | Protein Conformation | Glycoproteins - chemistry | Child | Iduronidase - genetics | Sequence Deletion - genetics | Enzymes | Mucopolysaccharidosis | Hydrogen | Analysis | Mutation
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9. Full Text Genotype–phenotype relationship in mucopolysaccharidosis II: predictive power of IDS variants for the neuronopathic phenotype
by Vollebregt, Audrey A M and Hoogeveen‐Westerveld, Marianne and Kroos, Marian A and Oussoren, Esmee and Plug, Iris and Ruijter, George J and van der Ploeg, Ans T and Pijnappel, W W M Pim
Developmental Medicine & Child Neurology, ISSN 0012-1622, 10/2017, Volume 59, Issue 10, pp. 1063 - 1070
Aim Mucopolysaccharidosis type II (MPS II) is caused by variants in the iduronate‐2‐sulphatase gene (IDS). Patients can be either neuronopathic with... 
IDURONATE-2-SULFATASE GENE | MOLECULAR CHARACTERIZATION | PROTEIN | STRUCTURAL-ANALYSIS | PEDIATRICS | HUNTER-DISEASE | MUTATIONS | IDENTIFICATION | EXPRESSION | CLINICAL NEUROLOGY | Intelligence | Humans | Middle Aged | Glycoproteins - metabolism | Child, Preschool | Immunoblotting | Educational Status | Genetic Variation | Young Adult | Netherlands | Mucopolysaccharidosis II - metabolism | Mass Spectrometry | Mucopolysaccharidosis II - genetics | Adult | Epilepsy - genetics | Child | Glycosaminoglycans - urine | Epilepsy - enzymology | Glycoproteins - genetics | Epilepsy - psychology | Genetic Association Studies | Phenotype | Mucopolysaccharidosis II - drug therapy | Adolescent | Mucopolysaccharidosis II - psychology | Cohort Studies
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10. Full Text Cell cycle is disturbed in mucopolysaccharidosis type II fibroblasts, and can be improved by genistein
by Moskot, Marta and Gabig-Cimińska, Magdalena and Jakóbkiewicz-Banecka, Joanna and Węsierska, Magdalena and Bocheńska, Katarzyna and Węgrzyn, Grzegorz
Gene, ISSN 0378-1119, 07/2016, Volume 585, Issue 1, pp. 100 - 103
Mucopolysaccharidoses (MPSs) are inherited metabolic diseases caused by mutations resulting in deficiency of one of enzymes involved in degradation of... 
Fibroblasts | Genistein | Mucopolysaccharidoses | Cell cycle | Cell Division - genetics | Cell Cycle - genetics | Cell Line | Glycosaminoglycans - metabolism | Humans | Genistein - pharmacology | Mucopolysaccharidosis II - genetics | Mucopolysaccharidosis I - genetics | Cell Proliferation - drug effects | Fibroblasts - cytology | Cell Cycle - drug effects | Physiological aspects | Mucopolysaccharidosis | Angiogenesis inhibitors | Isoflavones | Enzymes | Analysis | Hyaluronic acid | Sulfates
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11. Full Text Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)
by Martin, Rick and Beck, Michael and Eng, Christine and Giugliani, Roberto and Harmatz, Paul and Munoz, Veronica and Muenzer, Joseph
Pediatrics, ISSN 0031-4005, 02/2008, Volume 121, Issue 2, pp. e377 - e386
Mucopolysaccharidosis II, also known as Hunter syndrome, is a rare, X-linked disorder caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase,... 
Lysosomal storage disease | CARDIOVASCULAR CHANGES | lysosomal storage disease | BONE-MARROW-TRANSPLANTATION | SYNDROME MPS II | TERM-FOLLOW-UP | PEDIATRICS | CARPAL-TUNNEL-SYNDROME | AIRWAY-OBSTRUCTION | LYSOSOMAL STORAGE DISORDERS | ENZYME REPLACEMENT THERAPY | DRIED BLOOD SPOTS | PRENATAL-DIAGNOSIS | Diagnosis, Differential | Pedigree | History, 20th Century | Humans | Mucopolysaccharidosis II - therapy | Mucopolysaccharidosis II - genetics | Mucopolysaccharidosis II - history | Female | Male | Mucopolysaccharidosis II - diagnosis | Enzymes | Pediatrics | Medical diagnosis | Neurological disorders
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12. Full Text Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC/MS/MS
by Tanaka, Noboru and Kida, Sachiho and Kinoshita, Masafumi and Morimoto, Hideto and Shibasaki, Tadao and Tachibana, Katsuhiko and Yamamoto, Ryuji
Molecular Genetics and Metabolism, ISSN 1096-7192, 09/2018, Volume 125, Issue 1-2, pp. 53 - 58
Mucopolysaccharidosis type II (MPS II or Hunter syndrome) is a lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS), an enzyme that... 
Mucopolysaccharidosis type II | Glycosaminoglycan | Iduronate-2-sulfatase | Cerebrospinal fluid | Enzyme replacement therapy | LC/MS/MS | MEDICINE, RESEARCH & EXPERIMENTAL | GLYCOSAMINOGLYCANS | DISORDERS | MASS-SPECTROMETRY | CHILDREN | HUNTER-SYNDROME | ENDOCRINOLOGY & METABOLISM | GENETICS & HEREDITY | Humans | Blood-Brain Barrier - drug effects | Mice, Knockout | Brain - drug effects | Brain - metabolism | Mucopolysaccharidosis II - pathology | Tandem Mass Spectrometry | Heparitin Sulfate - cerebrospinal fluid | Animals | Mucopolysaccharidosis II - cerebrospinal fluid | Mucopolysaccharidosis II - drug therapy | Heparitin Sulfate - genetics | Nervous System Diseases - cerebrospinal fluid | Brain - pathology | Chromatography, Liquid | Iduronate Sulfatase - genetics | Mucopolysaccharidosis II - genetics | Mice | Biomarkers - cerebrospinal fluid | Receptors, Transferrin - genetics | Dermatan Sulfate - cerebrospinal fluid | Disease Models, Animal | Nervous System Diseases - pathology | Enzymes | Medical research | Glycosaminoglycans | Analysis | Medicine, Experimental | Mucopolysaccharidosis | Liquid chromatography | Sulfates | Mass spectrometry
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13. Full Text A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II
by Sonoda, Hiroyuki and Morimoto, Hideto and Yoden, Eiji and Koshimura, Yuri and Kinoshita, Masafumi and Golovina, Galina and Takagi, Haruna and Yamamoto, Ryuji and Minami, Kohtaro and Mizoguchi, Akira and Tachibana, Katsuhiko and Hirato, Tohru and Takahashi, Kenichi
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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14. Full Text Sangamo's landmark genome editing trial gets mixed reception
by Sheridan, C
NATURE BIOTECHNOLOGY, ISSN 1087-0156, 10/2018, Volume 36, Issue 10, pp. 907 - 908
BIOTECHNOLOGY & APPLIED MICROBIOLOGY | Biotechnology | Mucopolysaccharidosis II - enzymology | Humans | CRISPR-Cas Systems | Mucopolysaccharidosis II - therapy | Mucopolysaccharidosis II - genetics | Clinical Trials as Topic | Gene Editing | Zinc Finger Nucleases - genetics | Genetic engineering | Product development | Biotechnology industry | Genomes
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