387 related articles for article (PubMed ID: 19621331)
1. Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression.
Sun B; Zhang H; Bird A; Li S; Young SP; Koeberl DD
J Gene Med; 2009 Oct; 11(10):913-20. PubMed ID: 19621331
[TBL] [Abstract][Full Text] [Related]
2. Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.
Sun B; Li S; Bird A; Yi H; Kemper A; Thurberg BL; Koeberl DD
J Gene Med; 2010 Nov; 12(11):881-91. PubMed ID: 20967919
[TBL] [Abstract][Full Text] [Related]
3. Adjunctive β2-agonist treatment reduces glycogen independently of receptor-mediated acid α-glucosidase uptake in the limb muscles of mice with Pompe disease.
Farah BL; Madden L; Li S; Nance S; Bird A; Bursac N; Yen PM; Young SP; Koeberl DD
FASEB J; 2014 May; 28(5):2272-80. PubMed ID: 24448824
[TBL] [Abstract][Full Text] [Related]
4. Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.
Cagin U; Puzzo F; Gomez MJ; Moya-Nilges M; Sellier P; Abad C; Van Wittenberghe L; Daniele N; Guerchet N; Gjata B; Collaud F; Charles S; Sola MS; Boyer O; Krijnse-Locker J; Ronzitti G; Colella P; Mingozzi F
Mol Ther; 2020 Sep; 28(9):2056-2072. PubMed ID: 32526204
[TBL] [Abstract][Full Text] [Related]
5. Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa
Sellier P; Vidal P; Bertin B; Gicquel E; Bertil-Froidevaux E; Georger C; van Wittenberghe L; Miranda A; Daniele N; Richard I; Gross DA; Mingozzi F; Collaud F; Ronzitti G
J Inherit Metab Dis; 2024 Jan; 47(1):119-134. PubMed ID: 37204237
[TBL] [Abstract][Full Text] [Related]
6. Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease.
Han SO; Gheorghiu D; Chang A; Mapatano SH; Li S; Brooks E; Koeberl D
Hum Gene Ther; 2022 May; 33(9-10):479-491. PubMed ID: 35081735
[TBL] [Abstract][Full Text] [Related]
7. Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy.
Sun B; Young SP; Li P; Di C; Brown T; Salva MZ; Li S; Bird A; Yan Z; Auten R; Hauschka SD; Koeberl DD
Mol Ther; 2008 Aug; 16(8):1366-71. PubMed ID: 18560415
[TBL] [Abstract][Full Text] [Related]
8. Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease.
Zhang P; Sun B; Osada T; Rodriguiz R; Yang XY; Luo X; Kemper AR; Clay TM; Koeberl DD
Hum Gene Ther; 2012 May; 23(5):460-72. PubMed ID: 22260439
[TBL] [Abstract][Full Text] [Related]
9. Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
Keeler AM; Zieger M; Todeasa SH; McCall AL; Gifford JC; Birsak S; Choudhury SR; Byrne BJ; Sena-Esteves M; ElMallah MK
Hum Gene Ther; 2019 Jan; 30(1):57-68. PubMed ID: 29901418
[TBL] [Abstract][Full Text] [Related]
10. 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]
11. Salmeterol enhances the cardiac response to gene therapy in Pompe disease.
Han SO; Li S; Koeberl DD
Mol Genet Metab; 2016 May; 118(1):35-40. PubMed ID: 27017193
[TBL] [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; 12(5):876-84. PubMed ID: 16005263
[TBL] [Abstract][Full Text] [Related]
13. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
Puzzo F; Colella P; Biferi MG; Bali D; Paulk NK; Vidal P; Collaud F; Simon-Sola M; Charles S; Hardet R; Leborgne C; Meliani A; Cohen-Tannoudji M; Astord S; Gjata B; Sellier P; van Wittenberghe L; Vignaud A; Boisgerault F; Barkats M; Laforet P; Kay MA; Koeberl DD; Ronzitti G; Mingozzi F
Sci Transl Med; 2017 Nov; 9(418):. PubMed ID: 29187643
[TBL] [Abstract][Full Text] [Related]
14. Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.
Han SO; Li S; Brooks ED; Masat E; Leborgne C; Banugaria S; Bird A; Mingozzi F; Waldmann H; Koeberl D
Hum Gene Ther; 2015 Jan; 26(1):26-35. PubMed ID: 25382056
[TBL] [Abstract][Full Text] [Related]
15. Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.
Sun B; Zhang H; Benjamin DK; Brown T; Bird A; Young SP; McVie-Wylie A; Chen YT; Koeberl DD
Mol Ther; 2006 Dec; 14(6):822-30. PubMed ID: 16987711
[TBL] [Abstract][Full Text] [Related]
16. Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.
Han SO; Li S; Bird A; Koeberl D
Hum Gene Ther; 2015 Nov; 26(11):743-50. PubMed ID: 26417913
[TBL] [Abstract][Full Text] [Related]
17. Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease.
Han SO; Gheorghiu D; Li S; Kang HR; Koeberl D
Hum Gene Ther; 2022 May; 33(9-10):492-498. PubMed ID: 35102744
[TBL] [Abstract][Full Text] [Related]
18. 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]
19. 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]
20. Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors.
Fraites TJ; Schleissing MR; Shanely RA; Walter GA; Cloutier DA; Zolotukhin I; Pauly DF; Raben N; Plotz PH; Powers SK; Kessler PD; Byrne BJ
Mol Ther; 2002 May; 5(5 Pt 1):571-8. PubMed ID: 11991748
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]