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556 related items for PubMed ID: 25956830

  • 1. Enzyme replacement therapy of a novel humanized mouse model of globoid cell leukodystrophy.
    Matthes F, Andersson C, Stein A, Eistrup C, Fogh J, Gieselmann V, Wenger DA, Matzner U.
    Exp Neurol; 2015 Sep; 271():36-45. PubMed ID: 25956830
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  • 2. Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy.
    Hawkins-Salsbury JA, Shea L, Jiang X, Hunter DA, Guzman AM, Reddy AS, Qin EY, Li Y, Gray SJ, Ory DS, Sands MS.
    J Neurosci; 2015 Apr 22; 35(16):6495-505. PubMed ID: 25904800
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  • 3. CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model.
    Lin DS, Hsiao CD, Liau I, Lin SP, Chiang MF, Chuang CK, Wang TJ, Wu TY, Jian YR, Huang SF, Liu HL.
    Mol Genet Metab; 2011 Aug 22; 103(4):367-77. PubMed ID: 21620749
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  • 4. Mitigation of cerebellar neuropathy in globoid cell leukodystrophy mice by AAV-mediated gene therapy.
    Lin DS, Hsiao CD, Lee AY, Ho CS, Liu HL, Wang TJ, Jian YR, Hsu JC, Huang ZD, Lee TH, Chiang MF.
    Gene; 2015 Oct 15; 571(1):81-90. PubMed ID: 26115766
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  • 5. Transplantation of mouse embryonic stem cell-derived oligodendrocytes in the murine model of globoid cell leukodystrophy.
    Kuai XL, Ni RZ, Zhou GX, Mao ZB, Zhang JF, Yi N, Liu ZX, Shao N, Ni WK, Wang ZW.
    Stem Cell Res Ther; 2015 Mar 14; 6(1):30. PubMed ID: 25888852
    [Abstract] [Full Text] [Related]

  • 6. Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy.
    Qin EY, Hawkins-Salsbury JA, Jiang X, Reddy AS, Farber NB, Ory DS, Sands MS.
    Mol Genet Metab; 2012 Sep 14; 107(1-2):186-96. PubMed ID: 22704480
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  • 8. AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy.
    Rafi MA, Zhi Rao H, Passini MA, Curtis M, Vanier MT, Zaka M, Luzi P, Wolfe JH, Wenger DA.
    Mol Ther; 2005 May 14; 11(5):734-44. PubMed ID: 15851012
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  • 9. AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.
    Lin D, Fantz CR, Levy B, Rafi MA, Vogler C, Wenger DA, Sands MS.
    Mol Ther; 2005 Sep 14; 12(3):422-30. PubMed ID: 15996520
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  • 10. Characterization and application of a disease-cell model for a neurodegenerative lysosomal disease.
    Ribbens JJ, Moser AB, Hubbard WC, Bongarzone ER, Maegawa GH.
    Mol Genet Metab; 2014 Feb 14; 111(2):172-83. PubMed ID: 24094551
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  • 11. Intrinsic resistance of neural stem cells to toxic metabolites may make them well suited for cell non-autonomous disorders: evidence from a mouse model of Krabbe leukodystrophy.
    Taylor RM, Lee JP, Palacino JJ, Bower KA, Li J, Vanier MT, Wenger DA, Sidman RL, Snyder EY.
    J Neurochem; 2006 Jun 14; 97(6):1585-99. PubMed ID: 16805770
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  • 12. Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction.
    Weinstock NI, Shin D, Dhimal N, Hong X, Irons EE, Silvestri NJ, Reed CB, Nguyen D, Sampson O, Cheng YC, Lau JTY, Bongarzone ER, Kofler J, Escolar ML, Gelb MH, Wrabetz L, Feltri ML.
    Neuron; 2020 Jul 08; 107(1):65-81.e9. PubMed ID: 32375064
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  • 13. Generation of a mouse with low galactocerebrosidase activity by gene targeting: a new model of globoid cell leukodystrophy (Krabbe disease).
    Luzi P, Rafi MA, Zaka M, Curtis M, Vanier MT, Wenger DA.
    Mol Genet Metab; 2001 Jul 08; 73(3):211-23. PubMed ID: 11461188
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  • 14. Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features.
    Rafi MA, Rao HZ, Luzi P, Luddi A, Curtis MT, Wenger DA.
    Mol Genet Metab; 2015 Mar 08; 114(3):459-66. PubMed ID: 25533112
    [Abstract] [Full Text] [Related]

  • 15. Insights into the Pathogenesis and Treatment of Krabbe Disease.
    Bongarzone ER, Escolar ML, Gray SJ, Kafri T, Vite CH, Sands MS.
    Pediatr Endocrinol Rev; 2016 Jun 08; 13 Suppl 1():689-96. PubMed ID: 27491217
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