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Items: 1 to 20 of 78

1.

Airway smooth muscle dysfunction in Pompe (Gaa-/- ) mice.

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 1;312(6):L873-L881. doi: 10.1152/ajplung.00568.2016. Epub 2017 Mar 23.

PMID:
28336814
2.

Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology.

Turner SMF, Falk DJ, Byrne BJ, Fuller DD.

Physiol Genomics. 2016 Nov 1;48(11):785-794. doi: 10.1152/physiolgenomics.00075.2016. Epub 2016 Sep 9.

PMID:
27614205
3.

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. doi: 10.1016/j.ymgme.2016.03.006. Epub 2016 Mar 18.

4.

Peripheral nerve and neuromuscular junction pathology in Pompe disease.

Falk DJ, Todd AG, Lee S, Soustek MS, ElMallah MK, Fuller DD, Notterpek L, Byrne BJ.

Hum Mol Genet. 2015 Feb 1;24(3):625-36. doi: 10.1093/hmg/ddu476. Epub 2014 Sep 12.

5.

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 Jr, 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 Jul 18;9(7):e102092. doi: 10.1371/journal.pone.0102092. eCollection 2014.

6.

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent.

Nayak S, Doerfler PA, Porvasnik SL, Cloutier DD, Khanna R, Valenzano KJ, Herzog RW, Byrne BJ.

PLoS One. 2014 Jun 4;9(6):e98336. doi: 10.1371/journal.pone.0098336. eCollection 2014.

7.

Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease.

Nilsson MI, Samjoo IA, Hettinga BP, Koeberl DD, Zhang H, Hawke TJ, Nissar AA, Ali T, Brandt L, Ansari MU, Hazari H, Patel N, Amon J, Tarnopolsky MA.

Mol Genet Metab. 2012 Nov;107(3):469-79. doi: 10.1016/j.ymgme.2012.09.010. Epub 2012 Sep 15.

PMID:
23041258
8.

Modulation of starch digestion for slow glucose release through "toggling" of activities of mucosal α-glucosidases.

Lee BH, Eskandari R, Jones K, Reddy KR, Quezada-Calvillo R, Nichols BL, Rose DR, Hamaker BR, Pinto BM.

J Biol Chem. 2012 Sep 14;287(38):31929-38. doi: 10.1074/jbc.M112.351858. Epub 2012 Jul 31.

9.

The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.

Khanna R, Flanagan JJ, Feng J, Soska R, Frascella M, Pellegrino LJ, Lun Y, Guillen D, Lockhart DJ, Valenzano KJ.

PLoS One. 2012;7(7):e40776. doi: 10.1371/journal.pone.0040776. Epub 2012 Jul 18.

10.

Mapping the T helper cell response to acid α-glucosidase in Pompe mice.

Nayak S, Sivakumar R, Cao O, Daniell H, Byrne BJ, Herzog RW.

Mol Genet Metab. 2012 Jun;106(2):189-95. doi: 10.1016/j.ymgme.2012.03.009. Epub 2012 Mar 23.

11.

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype.

van Til NP, Stok M, Aerts Kaya FS, de Waard MC, Farahbakhshian E, Visser TP, Kroos MA, Jacobs EH, Willart MA, van der Wegen P, Scholte BJ, Lambrecht BN, Duncker DJ, van der Ploeg AT, Reuser AJ, Verstegen MM, Wagemaker G.

Blood. 2010 Jul 1;115(26):5329-37. doi: 10.1182/blood-2009-11-252874. Epub 2010 Apr 12.

12.

Impaired organization and function of myofilaments in single muscle fibers from a mouse model of Pompe disease.

Xu S, Galperin M, Melvin G, Horowits R, Raben N, Plotz P, Yu L.

J Appl Physiol (1985). 2010 May;108(5):1383-8. doi: 10.1152/japplphysiol.01253.2009. Epub 2010 Mar 11.

13.

Hematopoietic contribution to skeletal muscle regeneration in acid alpha-glucosidase knockout mice.

Mori J, Ishihara Y, Matsuo K, Nakajima H, Terada N, Kosaka K, Kizaki Z, Sugimoto T.

J Histochem Cytochem. 2008 Sep;56(9):811-7. doi: 10.1369/jhc.2008.951244. Epub 2008 May 27.

14.

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model.

Joseph A, Munroe K, Housman M, Garman R, Richards S.

Clin Exp Immunol. 2008 Apr;152(1):138-46. doi: 10.1111/j.1365-2249.2008.03602.x. Epub 2008 Feb 25.

15.

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.

Sun B, Bird A, Young SP, Kishnani PS, Chen YT, Koeberl DD.

Am J Hum Genet. 2007 Nov;81(5):1042-9. Epub 2007 Sep 21.

16.

Pompe disease: current state of treatment modalities and animal models.

Geel TM, McLaughlin PM, de Leij LF, Ruiters MH, Niezen-Koning KE.

Mol Genet Metab. 2007 Dec;92(4):299-307. Epub 2007 Sep 7. Review.

PMID:
17826266
17.

Differential muscular glycogen clearance after enzyme replacement therapy in a mouse model of Pompe disease.

Hawes ML, Kennedy W, O'Callaghan MW, Thurberg BL.

Mol Genet Metab. 2007 Aug;91(4):343-51. Epub 2007 Jun 14.

PMID:
17572127
18.

Optimized preservation of CNS morphology for the identification of glycogen in the Pompe mouse model.

Taksir TV, Griffiths D, Johnson J, Ryan S, Shihabuddin LS, Thurberg BL.

J Histochem Cytochem. 2007 Oct;55(10):991-8. Epub 2007 May 17.

PMID:
17510371
19.

Hyaluronidase increases the biodistribution of acid alpha-1,4 glucosidase in the muscle of Pompe disease mice: an approach to enhance the efficacy of enzyme replacement therapy.

Matalon R, Surendran S, Campbell GA, Michals-Matalon K, Tyring SK, Grady J, Cheng S, Kaye E.

Biochem Biophys Res Commun. 2006 Nov 24;350(3):783-7. Epub 2006 Oct 2.

PMID:
17027913
20.

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