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634 related items for PubMed ID: 19263466

  • 1. Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of alpha-glucosidase in Pompe disease.
    Douillard-Guilloux G, Richard E, Batista L, Caillaud C.
    J Gene Med; 2009 Apr; 11(4):279-87. PubMed ID: 19263466
    [Abstract] [Full Text] [Related]

  • 2. Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.
    Xu F, Ding E, Liao SX, Migone F, Dai J, Schneider A, Serra D, Chen YT, Amalfitano A.
    Gene Ther; 2004 Nov; 11(21):1590-8. PubMed ID: 15356673
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  • 3. Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.
    Sun B, Zhang H, Franco LM, Brown T, Bird A, Schneider A, Koeberl DD.
    Mol Ther; 2005 Jun; 11(6):889-98. PubMed ID: 15922959
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  • 4. Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II.
    Douillard-Guilloux G, Raben N, Takikita S, Batista L, Caillaud C, Richard E.
    Hum Mol Genet; 2008 Dec 15; 17(24):3876-86. PubMed ID: 18782850
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  • 5. Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors.
    Mah C, Cresawn KO, Fraites TJ, Pacak CA, Lewis MA, Zolotukhin I, Byrne BJ.
    Gene Ther; 2005 Sep 15; 12(18):1405-9. PubMed ID: 15920463
    [Abstract] [Full Text] [Related]

  • 6. Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme.
    Raben N, Jatkar T, Lee A, Lu N, Dwivedi S, Nagaraju K, Plotz PH.
    Mol Ther; 2002 Nov 15; 6(5):601-8. PubMed ID: 12409258
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  • 9. Hyaluronidase increases the biodistribution of acid alpha-1,4 glucosidase in the muscle of Pompe disease mice: an approach to enhance the efficacy of enzyme replacement therapy.
    Matalon R, Surendran S, Campbell GA, Michals-Matalon K, Tyring SK, Grady J, Cheng S, Kaye E.
    Biochem Biophys Res Commun; 2006 Nov 24; 350(3):783-7. PubMed ID: 17027913
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  • 11. 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 24; 12(5):876-84. PubMed ID: 16005263
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  • 12. Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.
    Raben N, Fukuda T, Gilbert AL, de Jong D, Thurberg BL, Mattaliano RJ, Meikle P, Hopwood JJ, Nagashima K, Nagaraju K, Plotz PH.
    Mol Ther; 2005 Jan 24; 11(1):48-56. PubMed ID: 15585405
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  • 13. Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA.
    Xu F, Ding E, Migone F, Serra D, Schneider A, Chen YT, Amalfitano A.
    J Gene Med; 2005 Feb 24; 7(2):171-8. PubMed ID: 15515143
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  • 14. Correction of glycogen storage disease type II by enzyme replacement with a recombinant human acid maltase produced by over-expression in a CHO-DHFR(neg) cell line.
    Martiniuk F, Chen A, Donnabella V, Arvanitopoulos E, Slonim AE, Raben N, Plotz P, Rom WN.
    Biochem Biophys Res Commun; 2000 Oct 05; 276(3):917-23. PubMed ID: 11027569
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  • 15. 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 05; 9(9):554-63. PubMed ID: 11973631
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  • 16. Immortalization of murine muscle cells from lysosomal alpha-glucosidase deficient mice: a new tool to study pathophysiology and assess therapeutic strategies for Pompe disease.
    Douillard-Guilloux G, Mouly V, Caillaud C, Richard E.
    Biochem Biophys Res Commun; 2009 Oct 16; 388(2):333-8. PubMed ID: 19665008
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  • 17. Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.
    Sun B, Bird A, Young SP, Kishnani PS, Chen YT, Koeberl DD.
    Am J Hum Genet; 2007 Nov 16; 81(5):1042-9. PubMed ID: 17924344
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  • 18. 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 16; 11(1):57-65. PubMed ID: 15585406
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  • 20. New insights into therapeutic options for Pompe disease.
    Richard E, Douillard-Guilloux G, Caillaud C.
    IUBMB Life; 2011 Nov 16; 63(11):979-86. PubMed ID: 22002928
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