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PUBMED FOR HANDHELDS

Journal Abstract Search

257 related items for PubMed ID: 31060789

  • 21.
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  • 23. Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.
    Ma X, Liu Y, Tittiger M, Hennig A, Kovacs A, Popelka S, Wang B, Herati R, Bigg M, Ponder KP.
    Mol Ther; 2007 May; 15(5):889-902. PubMed ID: 17311010
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  • 25. Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow.
    Zheng Y, Rozengurt N, Ryazantsev S, Kohn DB, Satake N, Neufeld EF.
    Mol Genet Metab; 2003 Aug; 79(4):233-44. PubMed ID: 12948739
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  • 26. Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.
    Miyadera K, Conatser L, Llanga TA, Carlin K, O'Donnell P, Bagel J, Song L, Kurtzberg J, Samulski RJ, Gilger B, Hirsch ML.
    Mol Ther; 2020 Jun 03; 28(6):1455-1463. PubMed ID: 32330426
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  • 27. In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors.
    Di Natale P, Di Domenico C, Villani GR, Lombardo A, Follenzi A, Naldini L.
    Eur J Biochem; 2002 Jun 03; 269(11):2764-71. PubMed ID: 12047386
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  • 31. Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.
    Baldo G, Wozniak DF, Ohlemiller KK, Zhang Y, Giugliani R, Ponder KP.
    J Inherit Metab Dis; 2013 May 03; 36(3):499-512. PubMed ID: 22983812
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  • 34. TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage.
    Seclen E, Jang JC, Lawal AO, Pulicani S, Boyne A, Tkach D, Juillerat A, Duchateau P, Valton J.
    Mol Ther; 2024 Jun 05; 32(6):1643-1657. PubMed ID: 38582963
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  • 35. Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.
    El-Amouri SS, Dai M, Han JF, Brady RO, Pan D.
    Mol Ther; 2014 Dec 05; 22(12):2028-2037. PubMed ID: 25088464
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  • 36. Residual α-L-iduronidase activity in fibroblasts of mild to severe Mucopolysaccharidosis type I patients.
    Oussoren E, Keulemans J, van Diggelen OP, Oemardien LF, Timmermans RG, van der Ploeg AT, Ruijter GJ.
    Mol Genet Metab; 2013 Aug 05; 109(4):377-81. PubMed ID: 23786846
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  • 37. 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 05; 13(11):917-25. PubMed ID: 16482204
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  • 39. A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.
    Ou L, Przybilla MJ, Ahlat O, Kim S, Overn P, Jarnes J, O'Sullivan MG, Whitley CB.
    Mol Ther; 2020 Jun 03; 28(6):1442-1454. PubMed ID: 32278382
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  • 40. Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig.
    Ponder KP, Wang B, Wang P, Ma X, Herati R, Wang B, Cullen K, O'Donnell P, Ellinwood NM, Traas A, Primeau TM, Haskins ME.
    Mol Ther; 2006 Jul 03; 14(1):5-13. PubMed ID: 16698321
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