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Journal Abstract Search

179 related items for PubMed ID: 12443541

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  • 2. 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; 9(9):554-63. PubMed ID: 11973631
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  • 5. 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; 7(2):171-8. PubMed ID: 15515143
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  • 7. 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; 12(18):1405-9. PubMed ID: 15920463
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  • 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
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  • 9. 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; 11(1):48-56. PubMed ID: 15585405
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  • 12. Complete correction of acid alpha-glucosidase deficiency in Pompe disease fibroblasts in vitro, and lysosomally targeted expression in neonatal rat cardiac and skeletal muscle.
    Pauly DF, Johns DC, Matelis LA, Lawrence JH, Byrne BJ, Kessler PD.
    Gene Ther; 1998 Apr; 5(4):473-80. PubMed ID: 9614571
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  • 13. Twenty-two novel mutations in the lysosomal alpha-glucosidase gene (GAA) underscore the genotype-phenotype correlation in glycogen storage disease type II.
    Hermans MM, van Leenen D, Kroos MA, Beesley CE, Van Der Ploeg AT, Sakuraba H, Wevers R, Kleijer W, Michelakakis H, Kirk EP, Fletcher J, Bosshard N, Basel-Vanagaite L, Besley G, Reuser AJ.
    Hum Mutat; 2004 Jan; 23(1):47-56. PubMed ID: 14695532
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  • 15. Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy Pompe disease.
    Pauly DF, Fraites TJ, Toma C, Bayes HS, Huie ML, Hirschhorn R, Plotz PH, Raben N, Kessler PD, Byrne BJ.
    Hum Gene Ther; 2001 Mar 20; 12(5):527-38. PubMed ID: 11268285
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  • 16. Genetic defects in patients with glycogenosis type II (acid maltase deficiency).
    Raben N, Nichols RC, Boerkoel C, Plotz P.
    Muscle Nerve Suppl; 1995 Mar 20; 3():S70-4. PubMed ID: 7603531
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  • 17. 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 20; 12(5):876-84. PubMed ID: 16005263
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  • 18. Adenovirus-mediated transfer of the acid alpha-glucosidase gene into fibroblasts, myoblasts and myotubes from patients with glycogen storage disease type II leads to high level expression of enzyme and corrects glycogen accumulation.
    Nicolino MP, Puech JP, Kremer EJ, Reuser AJ, Mbebi C, Verdière-Sahuqué M, Kahn A, Poenaru L.
    Hum Mol Genet; 1998 Oct 20; 7(11):1695-702. PubMed ID: 9736771
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  • 19. [A retrospective study of six patients with late-onset Pompe disease].
    Saux A, Laforet P, Pagès AM, Figarella-Branger D, Pellissier JF, Pagès M, Labauge P.
    Rev Neurol (Paris); 2008 Apr 20; 164(4):336-42. PubMed ID: 18439925
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