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286 related items for PubMed ID: 12101432

  • 1. Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors.
    Beaty RM, Jackson M, Peterson D, Bird A, Brown T, Benjamin DK, Juopperi T, Kishnani P, Boney A, Chen YT, Koeberl DD.
    Gene Ther; 2002 Aug; 9(15):1015-22. PubMed ID: 12101432
    [Abstract] [Full Text] [Related]

  • 2. Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.
    Koeberl DD, Sun BD, Damodaran TV, Brown T, Millington DS, Benjamin DK, Bird A, Schneider A, Hillman S, Jackson M, Beaty RM, Chen YT.
    Gene Ther; 2006 Sep; 13(17):1281-9. PubMed ID: 16672983
    [Abstract] [Full Text] [Related]

  • 3. Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer.
    Ghosh A, Allamarvdasht M, Pan CJ, Sun MS, Mansfield BC, Byrne BJ, Chou JY.
    Gene Ther; 2006 Feb; 13(4):321-9. PubMed ID: 16195703
    [Abstract] [Full Text] [Related]

  • 4. Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia.
    Crane B, Luo X, Demaster A, Williams KD, Kozink DM, Zhang P, Brown TT, Pinto CR, Oka K, Sun F, Jackson MW, Chan L, Koeberl DD.
    Gene Ther; 2012 Apr; 19(4):443-52. PubMed ID: 21654821
    [Abstract] [Full Text] [Related]

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  • 6. Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia.
    Demaster A, Luo X, Curtis S, Williams KD, Landau DJ, Drake EJ, Kozink DM, Bird A, Crane B, Sun F, Pinto CR, Brown TT, Kemper AR, Koeberl DD.
    Hum Gene Ther; 2012 Apr; 23(4):407-18. PubMed ID: 22185325
    [Abstract] [Full Text] [Related]

  • 7. Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy.
    Brooks ED, Landau DJ, Everitt JI, Brown TT, Grady KM, Waskowicz L, Bass CR, D'Angelo J, Asfaw YG, Williams K, Kishnani PS, Koeberl DD.
    J Inherit Metab Dis; 2018 Nov; 41(6):965-976. PubMed ID: 30043186
    [Abstract] [Full Text] [Related]

  • 8. Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.
    Sun B, Zhang H, Franco LM, Young SP, Schneider A, Bird A, Amalfitano A, Chen YT, Koeberl DD.
    Mol Ther; 2005 Jan; 11(1):57-65. PubMed ID: 15585406
    [Abstract] [Full Text] [Related]

  • 9. Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia.
    Koeberl DD, Sun B, Bird A, Chen YT, Oka K, Chan L.
    Mol Ther; 2007 Jul; 15(7):1253-8. PubMed ID: 17505475
    [Abstract] [Full Text] [Related]

  • 10. The upstream enhancer elements of the G6PC promoter are critical for optimal G6PC expression in murine glycogen storage disease type Ia.
    Lee YM, Pan CJ, Koeberl DD, Mansfield BC, Chou JY.
    Mol Genet Metab; 2013 Nov; 110(3):275-80. PubMed ID: 23856420
    [Abstract] [Full Text] [Related]

  • 11. Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice.
    Sun MS, Pan CJ, Shieh JJ, Ghosh A, Chen LY, Mansfield BC, Ward JM, Byrne BJ, Chou JY.
    Hum Mol Genet; 2002 Sep 01; 11(18):2155-64. PubMed ID: 12189168
    [Abstract] [Full Text] [Related]

  • 12. Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.
    Franco LM, Sun B, Yang X, Bird A, Zhang H, Schneider A, Brown T, Young SP, Clay TM, Amalfitano A, Chen YT, Koeberl DD.
    Mol Ther; 2005 Nov 01; 12(5):876-84. PubMed ID: 16005263
    [Abstract] [Full Text] [Related]

  • 13. Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.
    Luo X, Hall G, Li S, Bird A, Lavin PJ, Winn MP, Kemper AR, Brown TT, Koeberl DD.
    Mol Ther; 2011 Nov 01; 19(11):1961-70. PubMed ID: 21730973
    [Abstract] [Full Text] [Related]

  • 14. Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy.
    Lee YM, Jun HS, Pan CJ, Lin SR, Wilson LH, Mansfield BC, Chou JY.
    Hepatology; 2012 Nov 01; 56(5):1719-29. PubMed ID: 22422504
    [Abstract] [Full Text] [Related]

  • 15. Glycogen storage disease type Ia mice with less than 2% of normal hepatic glucose-6-phosphatase-α activity restored are at risk of developing hepatic tumors.
    Kim GY, Lee YM, Kwon JH, Cho JH, Pan CJ, Starost MF, Mansfield BC, Chou JY.
    Mol Genet Metab; 2017 Mar 01; 120(3):229-234. PubMed ID: 28096054
    [Abstract] [Full Text] [Related]

  • 16. Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors.
    Raake PW, Hinkel R, Müller S, Delker S, Kreuzpointner R, Kupatt C, Katus HA, Kleinschmidt JA, Boekstegers P, Müller OJ.
    Gene Ther; 2008 Jan 01; 15(1):12-7. PubMed ID: 17943147
    [Abstract] [Full Text] [Related]

  • 17. An evolutionary approach to optimizing glucose-6-phosphatase-α enzymatic activity for gene therapy of glycogen storage disease type Ia.
    Zhang L, Cho JH, Arnaoutova I, Mansfield BC, Chou JY.
    J Inherit Metab Dis; 2019 May 01; 42(3):470-479. PubMed ID: 30714174
    [Abstract] [Full Text] [Related]

  • 18. Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors.
    Müller OJ, Leuchs B, Pleger ST, Grimm D, Franz WM, Katus HA, Kleinschmidt JA.
    Cardiovasc Res; 2006 Apr 01; 70(1):70-8. PubMed ID: 16448634
    [Abstract] [Full Text] [Related]

  • 19. Adeno-associated virus-mediated transfer of human acid maltase gene results in a transient reduction of glycogen accumulation in muscle of Japanese quail with acid maltase deficiency.
    Lin CY, Ho CH, Hsieh YH, Kikuchi T.
    Gene Ther; 2002 May 01; 9(9):554-63. PubMed ID: 11973631
    [Abstract] [Full Text] [Related]

  • 20. Mice expressing reduced levels of hepatic glucose-6-phosphatase-α activity do not develop age-related insulin resistance or obesity.
    Kim GY, Lee YM, Cho JH, Pan CJ, Jun HS, Springer DA, Mansfield BC, Chou JY.
    Hum Mol Genet; 2015 Sep 15; 24(18):5115-25. PubMed ID: 26089201
    [Abstract] [Full Text] [Related]

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