288 related articles for article (PubMed ID: 18782850)
1. 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; 17(24):3876-86. PubMed ID: 18782850
[TBL] [Abstract][Full Text] [Related]
2. 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
[TBL] [Abstract][Full Text] [Related]
3. 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
[TBL] [Abstract][Full Text] [Related]
4. 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
[TBL] [Abstract][Full Text] [Related]
5. 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
[TBL] [Abstract][Full Text] [Related]
6. Lentiviral vector delivery of shRNA into cultured primary myogenic cells: a tool for therapeutic target validation.
Richard E; Douillard-Guilloux G; Caillaud C
Methods Mol Biol; 2011; 709():223-35. PubMed ID: 21194031
[TBL] [Abstract][Full Text] [Related]
7. 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; 388(2):333-8. PubMed ID: 19665008
[TBL] [Abstract][Full Text] [Related]
8. Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease.
Douillard-Guilloux G; Raben N; Takikita S; Ferry A; Vignaud A; Guillet-Deniau I; Favier M; Thurberg BL; Roach PJ; Caillaud C; Richard E
Hum Mol Genet; 2010 Feb; 19(4):684-96. PubMed ID: 19959526
[TBL] [Abstract][Full Text] [Related]
9. 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
[TBL] [Abstract][Full Text] [Related]
10. 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
[TBL] [Abstract][Full Text] [Related]
11. 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
[TBL] [Abstract][Full Text] [Related]
12. 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
[TBL] [Abstract][Full Text] [Related]
13. 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
[TBL] [Abstract][Full Text] [Related]
14. 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; 12(5):876-84. PubMed ID: 16005263
[TBL] [Abstract][Full Text] [Related]
15. 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; 276(3):917-23. PubMed ID: 11027569
[TBL] [Abstract][Full Text] [Related]
16. Correction of glycogenosis type 2 by muscle-specific lentiviral vector.
Richard E; Douillard-Guilloux G; Batista L; Caillaud C
In Vitro Cell Dev Biol Anim; 2008; 44(10):397-406. PubMed ID: 18810562
[TBL] [Abstract][Full Text] [Related]
17. Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction.
Kyosen SO; Iizuka S; Kobayashi H; Kimura T; Fukuda T; Shen J; Shimada Y; Ida H; Eto Y; Ohashi T
Gene Ther; 2010 Apr; 17(4):521-30. PubMed ID: 20033064
[TBL] [Abstract][Full Text] [Related]
18. Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease.
Rucker M; Fraites TJ; Porvasnik SL; Lewis MA; Zolotukhin I; Cloutier DA; Byrne BJ
Development; 2004 Jun; 131(12):3007-19. PubMed ID: 15169761
[TBL] [Abstract][Full Text] [Related]
19. Pathological features of glycogen storage disease type II highlighted in the knockout mouse model.
Bijvoet AG; Van Hirtum H; Vermey M; Van Leenen D; Van Der Ploeg AT; Mooi WJ; Reuser AJ
J Pathol; 1999 Nov; 189(3):416-24. PubMed ID: 10547605
[TBL] [Abstract][Full Text] [Related]
20. Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.
Cresawn KO; Fraites TJ; Wasserfall C; Atkinson M; Lewis M; Porvasnik S; Liu C; Mah C; Byrne BJ
Hum Gene Ther; 2005 Jan; 16(1):68-80. PubMed ID: 15703490
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]