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Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease.

Zhang P, Luo X, Bird A, Li S, Koeberl DD.

Biores Open Access. 2012 Jun;1(3):109-14. doi: 10.1089/biores.2012.0217.


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.


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.


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.


Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.

Han SO, Ronzitti G, Arnson B, Leborgne C, Li S, Mingozzi F, Koeberl D.

Mol Ther Methods Clin Dev. 2017 Jan 11;4:126-136. doi: 10.1016/j.omtm.2016.12.010. eCollection 2017 Mar 17.


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.


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.


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.


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.


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.


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.


Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.

Sun B, Kulis MD, Young SP, Hobeika AC, Li S, Bird A, Zhang H, Li Y, Clay TM, Burks W, Kishnani PS, Koeberl DD.

Mol Ther. 2010 Feb;18(2):353-60. doi: 10.1038/mt.2009.195. Epub 2009 Aug 18.


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.


Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential.

Li Q, Miller R, Han PY, Pang J, Dinculescu A, Chiodo V, Hauswirth WW.

Mol Vis. 2008 Sep 24;14:1760-9.


Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease.

Wang G, Young SP, Bali D, Hutt J, Li S, Benson J, Koeberl DD.

Mol Ther Methods Clin Dev. 2014 Jun 11;1:14018. doi: 10.1038/mtm.2014.18. eCollection 2014. Erratum in: Mol Ther Methods Clin Dev. 2015;2:15002.


Immunomodulatory, liver depot gene therapy for Pompe disease.

Bond JE, Kishnani PS, Koeberl DD.

Cell Immunol. 2017 Dec 29. pii: S0008-8749(17)30238-1. doi: 10.1016/j.cellimm.2017.12.011. [Epub ahead of print]


Complement is an essential component of the immune response to adeno-associated virus vectors.

Zaiss AK, Cotter MJ, White LR, Clark SA, Wong NC, Holers VM, Bartlett JS, Muruve DA.

J Virol. 2008 Mar;82(6):2727-40. doi: 10.1128/JVI.01990-07. Epub 2008 Jan 16.


Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins.

Mays LE, Vandenberghe LH, Xiao R, Bell P, Nam HJ, Agbandje-McKenna M, Wilson JM.

J Immunol. 2009 May 15;182(10):6051-60. doi: 10.4049/jimmunol.0803965.


Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase.

Conlon TJ, Erger K, Porvasnik S, Cossette T, Roberts C, Combee L, Islam S, Kelley J, Cloutier D, Clément N, Abernathy CR, Byrne BJ.

Hum Gene Ther Clin Dev. 2013 Sep;24(3):127-33. doi: 10.1089/humc.2013.147.

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