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Items: 1 to 20 of 232


Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.

Ziegler RJ, Bercury SD, Fidler J, Zhao MA, Foley J, Taksir TV, Ryan S, Hodges BL, Scheule RK, Shihabuddin LS, Cheng SH.

Hum Gene Ther. 2008 Jun;19(6):609-21. doi: 10.1089/hum.2008.010.


Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors.

Fraites TJ Jr, 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.


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. doi: 10.1038/mt.2010.110. Epub 2010 Jun 15.


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. doi: 10.1002/jgm.1372.


Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease.

Todd AG, McElroy JA, Grange RW, Fuller DD, Walter GA, Byrne BJ, Falk DJ.

Ann Neurol. 2015 Aug;78(2):222-34. doi: 10.1002/ana.24433. Epub 2015 Jun 30.


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.


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. doi: 10.1089/hum.2011.063. Epub 2012 Mar 29.


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. doi: 10.1038/mt.2008.133. Epub 2008 Jun 17.


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.


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.


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.


Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase.

Amalfitano A, McVie-Wylie AJ, Hu H, Dawson TL, Raben N, Plotz P, Chen YT.

Proc Natl Acad Sci U S A. 1999 Aug 3;96(16):8861-6.


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. doi: 10.1002/jgm.1511. Epub 2010 Oct 22.


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. doi: 10.1038/mt.2013.96. Epub 2013 Jun 4.


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. doi: 10.1038/mt.2011.214. Epub 2011 Oct 18.


Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors.

Mah C, Cresawn KO, Fraites TJ Jr, Pacak CA, Lewis MA, Zolotukhin I, Byrne BJ.

Gene Ther. 2005 Sep;12(18):1405-9.


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. doi: 10.1089/hum.2014.115.


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. doi: 10.1096/fj.13-244202. Epub 2014 Jan 21.


Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease.

Sun B, Li S, Bird A, Koeberl DD.

Gene Ther. 2010 Dec;17(12):1500-5. doi: 10.1038/gt.2010.109. Epub 2010 Aug 5.


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. doi: 10.1089/hum.2015.033. Epub 2015 Sep 29.

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