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Journal Abstract Search

572 related items for PubMed ID: 30064964

  • 21.
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  • 22. Differences in MPS I and MPS II Disease Manifestations.
    Hampe CS, Yund BD, Orchard PJ, Lund TC, Wesley J, McIvor RS.
    Int J Mol Sci; 2021 Jul 23; 22(15):. PubMed ID: 34360653
    [Abstract] [Full Text] [Related]

  • 23. Glycosaminoglycans analysis in blood and urine of patients with mucopolysaccharidosis.
    Khan SA, Mason RW, Giugliani R, Orii K, Fukao T, Suzuki Y, Yamaguchi S, Kobayashi H, Orii T, Tomatsu S.
    Mol Genet Metab; 2018 Sep 23; 125(1-2):44-52. PubMed ID: 29779903
    [Abstract] [Full Text] [Related]

  • 24. Characterization of a HIR-Fab-IDS, Novel Iduronate 2-Sulfatase Fusion Protein for the Treatment of Neuropathic Mucopolysaccharidosis Type II (Hunter Syndrome).
    Gusarova VD, Smolov MA, Lyagoskin IV, Degterev MB, Rechetnik EV, Rodionov AV, Pantyushenko MS, Shukurov RR.
    BioDrugs; 2023 May 23; 37(3):375-395. PubMed ID: 37014547
    [Abstract] [Full Text] [Related]

  • 25. Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II.
    Garcia AR, DaCosta JM, Pan J, Muenzer J, Lamsa JC.
    Mol Genet Metab; 2007 Jun 23; 91(2):183-90. PubMed ID: 17459751
    [Abstract] [Full Text] [Related]

  • 26. Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy.
    Zhang H, Dickson PI, Stiles AR, Chen AH, Le SQ, McCaw P, Beasley J, Millington DS, Young SP.
    Clin Chim Acta; 2020 Sep 23; 508():179-184. PubMed ID: 32442432
    [Abstract] [Full Text] [Related]

  • 27. Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells.
    Smith MC, Belur LR, Karlen AD, Erlanson O, Podetz-Pedersen KM, McKenzie J, Detellis J, Gagnidze K, Parsons G, Robinson N, Labarre S, Shah S, Furcich J, Lund TC, Tsai HC, McIvor RS, Bonner M.
    Hum Gene Ther; 2022 Dec 23; 33(23-24):1279-1292. PubMed ID: 36226412
    [Abstract] [Full Text] [Related]

  • 28. Robust LC-MS/MS methods for analysis of heparan sulfate levels in CSF and brain for application in studies of MPS IIIA.
    Makower Å, Arnelöf E, Andersson T, Edlund PO, Gustavsson S, Janson J, Gelius SS, Tjernberg A.
    Bioanalysis; 2019 Aug 23; 11(15):1389-1403. PubMed ID: 31490106
    [Abstract] [Full Text] [Related]

  • 29. Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice.
    Hinderer C, Katz N, Louboutin JP, Bell P, Yu H, Nayal M, Kozarsky K, O'Brien WT, Goode T, Wilson JM.
    Hum Gene Ther; 2016 Nov 23; 27(11):906-915. PubMed ID: 27510804
    [Abstract] [Full Text] [Related]

  • 30. Validation of disaccharide compositions derived from dermatan sulfate and heparan sulfate in mucopolysaccharidoses and mucolipidoses II and III by tandem mass spectrometry.
    Tomatsu S, Montaño AM, Oguma T, Dung VC, Oikawa H, Gutiérrez ML, Yamaguchi S, Suzuki Y, Fukushi M, Barrera LA, Kida K, Kubota M, Orii T.
    Mol Genet Metab; 2010 Feb 23; 99(2):124-31. PubMed ID: 19932038
    [Abstract] [Full Text] [Related]

  • 31. Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy.
    Jung SC, Park ES, Choi EN, Kim CH, Kim SJ, Jin DK.
    Mol Cells; 2010 Jul 23; 30(1):13-8. PubMed ID: 20652491
    [Abstract] [Full Text] [Related]

  • 32. CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome).
    Motas S, Haurigot V, Garcia M, Marcó S, Ribera A, Roca C, Sánchez X, Sánchez V, Molas M, Bertolin J, Maggioni L, León X, Ruberte J, Bosch F.
    JCI Insight; 2016 Jun 16; 1(9):e86696. PubMed ID: 27699273
    [Abstract] [Full Text] [Related]

  • 33. Efficient analysis of urinary glycosaminoglycans by LC-MS/MS in mucopolysaccharidoses type I, II and VI.
    Auray-Blais C, Bhérer P, Gagnon R, Young SP, Zhang HH, An Y, Clarke JT, Millington DS.
    Mol Genet Metab; 2011 Jan 16; 102(1):49-56. PubMed ID: 20934363
    [Abstract] [Full Text] [Related]

  • 34. Changes in glycogen and glycosaminoglycan levels in hepatocytes of iduronate-2-sulfatase knockout mice before and after recombinant iduronate-2-sulfatase supplementation.
    Lee JH, Choe YH, Kim SJ, Paik KH, Jin DK.
    Yonsei Med J; 2011 Mar 16; 52(2):263-7. PubMed ID: 21319344
    [Abstract] [Full Text] [Related]

  • 35. Targeting Brain Disease in MPSII: Preclinical Evaluation of IDS-Loaded PLGA Nanoparticles.
    Rigon L, Salvalaio M, Pederzoli F, Legnini E, Duskey JT, D'Avanzo F, De Filippis C, Ruozi B, Marin O, Vandelli MA, Ottonelli I, Scarpa M, Tosi G, Tomanin R.
    Int J Mol Sci; 2019 Apr 24; 20(8):. PubMed ID: 31022913
    [Abstract] [Full Text] [Related]

  • 36. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data.
    Giugliani R, Martins AM, Okuyama T, Eto Y, Sakai N, Nakamura K, Morimoto H, Minami K, Yamamoto T, Yamaoka M, Ikeda T, So S, Tanizawa K, Sonoda H, Schmidt M, Sato Y.
    Int J Mol Sci; 2021 Oct 10; 22(20):. PubMed ID: 34681597
    [Abstract] [Full Text] [Related]

  • 37. Newborn screening and diagnosis of mucopolysaccharidoses.
    Tomatsu S, Fujii T, Fukushi M, Oguma T, Shimada T, Maeda M, Kida K, Shibata Y, Futatsumori H, Montaño AM, Mason RW, Yamaguchi S, Suzuki Y, Orii T.
    Mol Genet Metab; 2013 Oct 10; 110(1-2):42-53. PubMed ID: 23860310
    [Abstract] [Full Text] [Related]

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  • 39.
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  • 40. Establishment of the Effectiveness of Early Versus Late Stem Cell Gene Therapy in Mucopolysaccharidosis II for Treating Central Versus Peripheral Disease.
    Mandolfo O, Liao A, Singh E, O'leary C, Holley RJ, Bigger BW.
    Hum Gene Ther; 2024 Apr 10; 35(7-8):243-255. PubMed ID: 37427450
    [Abstract] [Full Text] [Related]

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