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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.


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.


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.


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.


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.


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 Jr, Brown T, Bird A, Young SP, McVie-Wylie A, Chen YT, Koeberl DD.

Mol Ther. 2006 Dec;14(6):822-30. Epub 2006 Sep 20.


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.


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.


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.


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.


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.


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. Epub 2005 Jul 6.


Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.

Sun BD, Chen YT, Bird A, Amalfitano A, Koeberl DD.

Mol Ther. 2003 Feb;7(2):193-201.


Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.

Elmallah MK, Falk DJ, Nayak S, Federico RA, Sandhu MS, Poirier A, Byrne BJ, Fuller DD.

Mol Ther. 2014 Apr;22(4):702-12. doi: 10.1038/mt.2013.282. Epub 2013 Dec 12.


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.


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.


Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning.

Corti M, Cleaver B, Clément N, Conlon TJ, Faris KJ, Wang G, Benson J, Tarantal AF, Fuller D, Herzog RW, Byrne BJ.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):185-93. doi: 10.1089/humc.2015.068.


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.


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