375 related articles for article (PubMed ID: 28336814)
1. Airway smooth muscle dysfunction in Pompe (
Keeler AM; Liu D; Zieger M; Xiong L; Salemi J; Bellvé K; Byrne BJ; Fuller DD; ZhuGe R; ElMallah MK
Am J Physiol Lung Cell Mol Physiol; 2017 Jun; 312(6):L873-L881. PubMed ID: 28336814
[TBL] [Abstract][Full Text] [Related]
2. GAA deficiency disrupts distal airway cells in Pompe disease.
El Haddad L; Lai E; Murthy PKL; Biswas DD; Soufny R; Roger AL; Tata PR; ElMallah MK
Am J Physiol Lung Cell Mol Physiol; 2023 Sep; 325(3):L288-L298. PubMed ID: 37366541
[TBL] [Abstract][Full Text] [Related]
3. 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]
4. 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]
5. Remarkably low fibroblast acid α-glucosidase activity in three adults with Pompe disease.
Wens SC; Kroos MA; de Vries JM; Hoogeveen-Westerveld M; Wijgerde MG; van Doorn PA; van der Ploeg AT; Reuser AJ
Mol Genet Metab; 2012 Nov; 107(3):485-9. PubMed ID: 23000108
[TBL] [Abstract][Full Text] [Related]
6. Glycogen accumulation in smooth muscle of a Pompe disease mouse model.
McCall AL; Dhindsa JS; Bailey AM; Pucci LA; Strickland LM; ElMallah MK
J Smooth Muscle Res; 2021; 57(0):8-18. PubMed ID: 33883348
[TBL] [Abstract][Full Text] [Related]
7. Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle.
Koeberl DD; Luo X; Sun B; McVie-Wylie A; Dai J; Li S; Banugaria SG; Chen YT; Bali DS
Mol Genet Metab; 2011 Jun; 103(2):107-12. PubMed ID: 21397538
[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. Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease.
Liang Q; Vlaar EC; Pijnenburg JM; Rijkers E; Demmers JAA; Vulto AG; van der Ploeg AT; van Til NP; Pijnappel WWMP
J Proteomics; 2024 Jan; 291():105037. PubMed ID: 38288553
[TBL] [Abstract][Full Text] [Related]
10. 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]
11. 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]
12. Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology.
Turner SMF; Falk DJ; Byrne BJ; Fuller DD
Physiol Genomics; 2016 Nov; 48(11):785-794. PubMed ID: 27614205
[TBL] [Abstract][Full Text] [Related]
13. Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.
Qiu K; Falk DJ; Reier PJ; Byrne BJ; Fuller DD
Mol Ther; 2012 Jan; 20(1):21-7. PubMed ID: 22008916
[TBL] [Abstract][Full Text] [Related]
14. 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]
15. When more is less: excess and deficiency of autophagy coexist in skeletal muscle in Pompe disease.
Raben N; Baum R; Schreiner C; Takikita S; Mizushima N; Ralston E; Plotz P
Autophagy; 2009 Jan; 5(1):111-3. PubMed ID: 19001870
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. 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]
18. 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]
19. CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease.
Kan SH; Huang JY; Harb J; Rha A; Dalton ND; Christensen C; Chan Y; Davis-Turak J; Neumann J; Wang RY
Sci Rep; 2022 Dec; 12(1):21576. PubMed ID: 36517654
[TBL] [Abstract][Full Text] [Related]
20. Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease.
Wang J; Zhou CJ; Khodabukus A; Tran S; Han SO; Carlson AL; Madden L; Kishnani PS; Koeberl DD; Bursac N
Commun Biol; 2021 May; 4(1):524. PubMed ID: 33953320
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]