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Journal Abstract Search

415 related items for PubMed ID: 27510804

  • 1. 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; 27(11):906-915. PubMed ID: 27510804
    [Abstract] [Full Text] [Related]

  • 2. Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer.
    Laoharawee K, Podetz-Pedersen KM, Nguyen TT, Evenstar LB, Kitto KF, Nan Z, Fairbanks CA, Low WC, Kozarsky KF, McIvor RS.
    Hum Gene Ther; 2017 Aug; 28(8):626-638. PubMed ID: 28478695
    [Abstract] [Full Text] [Related]

  • 3. 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.
    Tanaka N, Kida S, Kinoshita M, Morimoto H, Shibasaki T, Tachibana K, Yamamoto R.
    Mol Genet Metab; 2018 Sep; 125(1-2):53-58. PubMed ID: 30064964
    [Abstract] [Full Text] [Related]

  • 4. Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy.
    Miwa S, Watabe AM, Shimada Y, Higuchi T, Kobayashi H, Fukuda T, Kato F, Ida H, Ohashi T.
    Mol Genet Metab; 2020 Aug; 130(4):262-273. PubMed ID: 32631737
    [Abstract] [Full Text] [Related]

  • 5. Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice.
    Watson G, Bastacky J, Belichenko P, Buddhikot M, Jungles S, Vellard M, Mobley WC, Kakkis E.
    Gene Ther; 2006 Jun; 13(11):917-25. PubMed ID: 16482204
    [Abstract] [Full Text] [Related]

  • 6. 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]

  • 7. Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II.
    Cho SY, Lee J, Ko AR, Kwak MJ, Kim S, Sohn YB, Park SW, Jin DK.
    Orphanet J Rare Dis; 2015 Oct 31; 10():141. PubMed ID: 26520066
    [Abstract] [Full Text] [Related]

  • 8. Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
    Hinderer C, Bell P, Louboutin JP, Zhu Y, Yu H, Lin G, Choa R, Gurda BL, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Tarantal AF, Casal ML, Haskins ME, Wilson JM.
    Mol Ther; 2015 Aug 31; 23(8):1298-1307. PubMed ID: 26022732
    [Abstract] [Full Text] [Related]

  • 9. Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II.
    Higuchi T, Shimizu H, Fukuda T, Kawagoe S, Matsumoto J, Shimada Y, Kobayashi H, Ida H, Ohashi T, Morimoto H, Hirato T, Nishino K, Eto Y.
    Mol Genet Metab; 2012 Sep 31; 107(1-2):122-8. PubMed ID: 22704483
    [Abstract] [Full Text] [Related]

  • 10. Gene Therapy for Mucopolysaccharidosis Type II-A Review of the Current Possibilities.
    Zapolnik P, Pyrkosz A.
    Int J Mol Sci; 2021 May 23; 22(11):. PubMed ID: 34070997
    [Abstract] [Full Text] [Related]

  • 11. Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.
    Belur LR, Temme A, Podetz-Pedersen KM, Riedl M, Vulchanova L, Robinson N, Hanson LR, Kozarsky KF, Orchard PJ, Frey WH, Low WC, McIvor RS.
    Hum Gene Ther; 2017 Jul 23; 28(7):576-587. PubMed ID: 28462595
    [Abstract] [Full Text] [Related]

  • 12. 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]

  • 13. The efficacy of intracerebroventricular idursulfase-beta enzyme replacement therapy in mucopolysaccharidosis II murine model: heparan sulfate in cerebrospinal fluid as a clinical biomarker of neuropathology.
    Sohn YB, Ko AR, Seong MR, Lee S, Kim MR, Cho SY, Kim JS, Sakaguchi M, Nakazawa T, Kosuga M, Seo JH, Okuyama T, Jin DK.
    J Inherit Metab Dis; 2018 Nov 23; 41(6):1235-1246. PubMed ID: 29978271
    [Abstract] [Full Text] [Related]

  • 14. Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice.
    Fu H, Kang L, Jennings JS, Moy SS, Perez A, Dirosario J, McCarty DM, Muenzer J.
    Gene Ther; 2007 Jul 23; 14(14):1065-77. PubMed ID: 17460717
    [Abstract] [Full Text] [Related]

  • 15. Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction.
    Podetz-Pedersen KM, Laoharawee K, Singh S, Nguyen TT, Smith MC, Temme A, Kozarsky K, McIvor RS, Belur LR.
    Hum Gene Ther; 2023 Jan 23; 34(1-2):8-18. PubMed ID: 36541357
    [Abstract] [Full Text] [Related]

  • 16. Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.
    Bhalla A, Ravi R, Fang M, Arguello A, Davis SS, Chiu CL, Blumenfeld JR, Nguyen HN, Earr TK, Wang J, Astarita G, Zhu Y, Fiore D, Scearce-Levie K, Diaz D, Cahan H, Troyer MD, Harris JM, Escolar ML.
    Int J Mol Sci; 2020 Jul 22; 21(15):. PubMed ID: 32707880
    [Abstract] [Full Text] [Related]

  • 17. Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys.
    Hordeaux J, Hinderer C, Buza EL, Louboutin JP, Jahan T, Bell P, Chichester JA, Tarantal AF, Wilson JM.
    Hum Gene Ther; 2019 Aug 22; 30(8):957-966. PubMed ID: 31017018
    [Abstract] [Full Text] [Related]

  • 18. Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
    Arguello A, Meisner R, Thomsen ER, Nguyen HN, Ravi R, Simms J, Lo I, Speckart J, Holtzman J, Gill TM, Chan D, Cheng Y, Chiu CL, Dugas JC, Fang M, Lopez IA, Solanoy H, Tsogtbaatar B, Zhu Y, Bhalla A, Henne KR, Henry AG, Delucchi A, Costanzo S, Harris JM, Diaz D, Scearce-Levie K, Sanchez PE.
    JCI Insight; 2021 Oct 08; 6(19):. PubMed ID: 34622797
    [Abstract] [Full Text] [Related]

  • 19. Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II.
    Wakabayashi T, Shimada Y, Akiyama K, Higuchi T, Fukuda T, Kobayashi H, Eto Y, Ida H, Ohashi T.
    Hum Gene Ther; 2015 Jun 08; 26(6):357-66. PubMed ID: 25761450
    [Abstract] [Full Text] [Related]

  • 20. Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease.
    Janson CG, Romanova LG, Leone P, Nan Z, Belur L, McIvor RS, Low WC.
    Neurosurgery; 2014 Jan 08; 74(1):99-111. PubMed ID: 24077583
    [Abstract] [Full Text] [Related]

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