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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. 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]
6. Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease. Schaaf GJ; van Gestel TJM; In 't Groen SLM; de Jong B; Boomaars B; Tarallo A; Cardone M; Parenti G; van der Ploeg AT; Pijnappel WWMP Acta Neuropathol Commun; 2018 Nov; 6(1):119. PubMed ID: 30404653 [TBL] [Abstract][Full Text] [Related]
7. Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. Falk DJ; Mah CS; Soustek MS; Lee KZ; Elmallah MK; Cloutier DA; Fuller DD; Byrne BJ Mol Ther; 2013 Sep; 21(9):1661-7. PubMed ID: 23732990 [TBL] [Abstract][Full Text] [Related]
8. Neural deficits contribute to respiratory insufficiency in Pompe disease. DeRuisseau LR; Fuller DD; Qiu K; DeRuisseau KC; Donnelly WH; Mah C; Reier PJ; Byrne BJ Proc Natl Acad Sci U S A; 2009 Jun; 106(23):9419-24. PubMed ID: 19474295 [TBL] [Abstract][Full Text] [Related]
9. The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease. Khanna R; Powe AC; Lun Y; Soska R; Feng J; Dhulipala R; Frascella M; Garcia A; Pellegrino LJ; Xu S; Brignol N; Toth MJ; Do HV; Lockhart DJ; Wustman BA; Valenzano KJ PLoS One; 2014; 9(7):e102092. PubMed ID: 25036864 [TBL] [Abstract][Full Text] [Related]
10. Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice. Foley JW; Bercury SD; Finn P; Cheng SH; Scheule RK; Ziegler RJ Mol Ther; 2010 Sep; 18(9):1584-91. PubMed ID: 20551907 [TBL] [Abstract][Full Text] [Related]
12. Suppression of mTORC1 activation in acid-α-glucosidase-deficient cells and mice is ameliorated by leucine supplementation. Shemesh A; Wang Y; Yang Y; Yang GS; Johnson DE; Backer JM; Pessin JE; Zong H Am J Physiol Regul Integr Comp Physiol; 2014 Nov; 307(10):R1251-9. PubMed ID: 25231351 [TBL] [Abstract][Full Text] [Related]
13. Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease. Peng J; Dalton J; Butt M; Tracy K; Kennedy D; Haroldsen P; Cahayag R; Zoog S; O'Neill CA; Tsuruda LS J Pharmacol Exp Ther; 2017 Feb; 360(2):313-323. PubMed ID: 27856936 [TBL] [Abstract][Full Text] [Related]
15. Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects. Colella P; Sellier P; Gomez MJ; Biferi MG; Tanniou G; Guerchet N; Cohen-Tannoudji M; Moya-Nilges M; van Wittenberghe L; Daniele N; Gjata B; Krijnse-Locker J; Collaud F; Simon-Sola M; Charles S; Cagin U; Mingozzi F EBioMedicine; 2020 Nov; 61():103052. PubMed ID: 33039711 [TBL] [Abstract][Full Text] [Related]
16. The respiratory neuromuscular system in Pompe disease. Fuller DD; ElMallah MK; Smith BK; Corti M; Lawson LA; Falk DJ; Byrne BJ Respir Physiol Neurobiol; 2013 Nov; 189(2):241-9. PubMed ID: 23797185 [TBL] [Abstract][Full Text] [Related]
17. Therapeutic efficacy of 3,4-Diaminopyridine phosphate on neuromuscular junction in Pompe disease. Bragato C; Blasevich F; Ingenito G; Mantegazza R; Maggi L Biomed Pharmacother; 2021 May; 137():111357. PubMed ID: 33724918 [TBL] [Abstract][Full Text] [Related]
18. 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]
19. Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease. Taylor KM; Meyers E; Phipps M; Kishnani PS; Cheng SH; Scheule RK; Moreland RJ PLoS One; 2013; 8(2):e56181. PubMed ID: 23457523 [TBL] [Abstract][Full Text] [Related]
20. 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] [Next] [New Search]