Baodong Sun
Associate Professor in Pediatrics
My overall research interests are finding effective treatment for human glycogen storage diseases (GSDs) and other inherited metabolic disorders. My current research focuses on identification of novel therapeutic targets and development of effective therapies for GSD II (Pompe disease), GSD III (Cori disease), and GSD IV (Andersen disease) using cellular and animal disease models. The main therapeutic approaches we are using in our pre-clinical studies include protein/enzyme therapy, AAV-mediated gene therapy, and substrate reduction therapy with small molecule drugs.
Office Hours
Current Appointments & Affiliations
- Associate Professor in Pediatrics, Pediatrics, Medical Genetics, Pediatrics 2021
Contact Information
- 905 Lasalle Street, GSRB1 Room 4044, Durham, NC 27710
- Box 103856 Med Ctr, Durham, NC 27710
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baodong.sun@duke.edu
(919) 681-8933
- Background
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Education, Training, & Certifications
- Ph.D., China Medical University (China) 1995
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Previous Appointments & Affiliations
- Associate Professor of Pediatrics, Pediatrics, Medical Genetics, Pediatrics 2015 - 2020
- Assistant Professor of Pediatrics, Pediatrics, Medical Genetics, Pediatrics 2011 - 2015
- Expertise
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Global Scholarship
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Research
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- Research
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Selected Grants
- Gene therapy for glycogen storage disease type III awarded by National Institutes of Health 2022 - 2027
- Stable therapy in Pompe disease through genome editing awarded by National Institutes of Health 2021 - 2026
- CHARACTERIZATION OF GSD IX ¿2 and GSD VI MOUSE MODELS awarded by Kriya Therapeutics 2022 - 2025
- AAV gene therapy for the treatment of Glycogen Storage Disease IX y2 awarded by Kriya Therapeutics 2022
- Optimized GAA (GAA) as potentially improved therapeutic for the treatment of Pompe disease awarded by Codexis, Inc. 2020 - 2022
- Gene therapy by Lipid Nanoparticle-Mediated Delivery of Agl Messenger RNA in GSD III dogs awarded by Ultragenyx Pharmaceutical 2017 - 2022
- Gene therapy for Adult polyglucosan body disease (APBD) awarded by University of Pennsylvania 2020 - 2021
- Feasibility of using Bortezomib-based immunosuppressive approach to deplete anti-AAV antibodies in mice awarded by Asklepios BioPharmaceutical, Inc. 2018 - 2020
- Activity and Biodistribution of the AAV2/8-LSPhGAA in GAA-knockout mice awarded by Actus Therapeutics 2018
- Preclinical study of use of SVP-Rapamycin to induce immune tolerance to ERT in Pompe disease mice awarded by Selecta Biosciences, Inc. 2017 - 2018
- Evaluation of the use of Myozyme for treatment of glycogen storage disease type III and IV in murine disease models awarded by Roivant Sciences, Inc. 2015 - 2018
- Liver-specific knockdown of M6PR with RNAi therapeutics to increase enzyme delivery to muscle tissues of Pompe disease mice during ERT awarded by Alnylam Pharmaceuticals 2018
- In vivo efficacy studies of Fab-GAA treatment for GSDII, III, and IV in murine disease models awarded by Valerion Therapeutics, Inc 2015 - 2017
- To test the ability of SVP to prevent immune response in Pompe disease mouse model. awarded by Selecta Biosciences, Inc. 2015 - 2016
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External Relationships
- Asklepios BioPharmaceutical, Inc
- Bayer
- Kriya Therapeutics, Inc.
- Publications & Artistic Works
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Selected Publications
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Academic Articles
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Choi, Su Jin, John S. Yi, Jeong-A Lim, Thomas F. Tedder, Dwight D. Koeberl, William Jeck, Ankit K. Desai, Amy Rosenberg, Baodong Sun, and Priya S. Kishnani. “Successful AAV8 readministration: Suppression of capsid-specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease.” J Gene Med, March 30, 2023, e3509. https://doi.org/10.1002/jgm.3509.Full Text Open Access Copy Link to Item
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Lim, Jeong-A, Priya S. Kishnani, and Baodong Sun. “Suppression of pullulanase-induced cytotoxic T cell response with a dual promoter in GSD IIIa mice.” Jci Insight 7, no. 23 (December 8, 2022). https://doi.org/10.1172/jci.insight.152970.Full Text Open Access Copy Link to Item
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Korlimarla, Aditi, Jeong-A Lim, Paul McIntosh, Kanecia Zimmerman, Baodong D. Sun, and Priya S. Kishnani. “New Insights into Gastrointestinal Involvement in Late-Onset Pompe Disease: Lessons Learned from Bench and Bedside.” J Clin Med 10, no. 15 (July 30, 2021). https://doi.org/10.3390/jcm10153395.Full Text Open Access Copy Link to Item
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Gibson, Rebecca A., Jeong-A Lim, Su Jin Choi, Leticia Flores, Lani Clinton, Deeksha Bali, Sarah Young, Aravind Asokan, Baodong Sun, and Priya S. Kishnani. “Characterization of liver GSD IX γ2 pathophysiology in a novel Phkg2-/- mouse model.” Mol Genet Metab 133, no. 3 (July 2021): 269–76. https://doi.org/10.1016/j.ymgme.2021.05.008.Full Text Open Access Copy Link to Item
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Korlimarla, Aditi, Jeong-A Lim, Paul McIntosh, Baodong Sun, and Priya S. Kishnani. “New insights into GI manifestations in late-onset Pompe disease: Lessons from the bench and bedside.” Molecular Genetics and Metabolism 132, no. 2 (February 2021): S58–59. https://doi.org/10.1016/j.ymgme.2020.12.130.Full Text Open Access Copy
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Lim, Jeong-A, Su Jin Choi, Fengqin Gao, Priya S. Kishnani, and Baodong Sun. “A Novel Gene Therapy Approach for GSD III Using an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme.” Mol Ther Methods Clin Dev 18 (September 11, 2020): 240–49. https://doi.org/10.1016/j.omtm.2020.05.034.Full Text Open Access Copy Link to Item
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Metzendorf, Christoph, Katharina Wineberger, Jenny Rausch, Antonio Cigliano, Kristin Peters, Baodong Sun, Daniela Mennerich, et al. “Transcriptomic and Proteomic Analysis of Clear Cell Foci (CCF) in the Human Non-Cirrhotic Liver Identifies Several Differentially Expressed Genes and Proteins with Functions in Cancer Cell Biology and Glycogen Metabolism.” Molecules 25, no. 18 (September 10, 2020). https://doi.org/10.3390/molecules25184141.Full Text Open Access Copy Link to Item
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Kishnani, Priya S., Baodong Sun, and Dwight D. Koeberl. “Gene therapy for glycogen storage diseases.” Hum Mol Genet 28, no. R1 (October 1, 2019): R31–41. https://doi.org/10.1093/hmg/ddz133.Full Text Open Access Copy Link to Item
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Korlimarla, Aditi, Jeong-A Lim, Priya S. Kishnani, and Baodong Sun. “An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond.” Ann Transl Med 7, no. 13 (July 2019): 289. https://doi.org/10.21037/atm.2019.04.49.Full Text Open Access Copy Link to Item
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Lim, Jeong-A, Haiqing Yi, Fengqin Gao, Nina Raben, Priya S. Kishnani, and Baodong Sun. “Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease.” Mol Ther Methods Clin Dev 12 (March 15, 2019): 233–45. https://doi.org/10.1016/j.omtm.2019.01.006.Full Text Open Access Copy Link to Item
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Korlimarla, A., S. Austin, B. Sun, and P. Kishnani. “Hepatic Manifestations in Glycogen Storage Disease Type III.” Current Pathobiology Reports 6, no. 4 (December 1, 2018): 233–40. https://doi.org/10.1007/s40139-018-0182-x.Full Text Open Access Copy
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Lim, Jeong-A, Baodong Sun, Rosa Puertollano, and Nina Raben. “Therapeutic Benefit of Autophagy Modulation in Pompe Disease.” Mol Ther 26, no. 7 (July 5, 2018): 1783–96. https://doi.org/10.1016/j.ymthe.2018.04.025.Full Text Open Access Copy Link to Item
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Lim, Han-Hyuk, Haiqing Yi, Takashi K. Kishimoto, Fengqin Gao, Baodong Sun, and Priya S. Kishnani. “A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease.” Mol Genet Metab Rep 13 (December 2017): 18–22. https://doi.org/10.1016/j.ymgmr.2017.03.005.Full Text Open Access Copy Link to Item
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Yi, Haiqing, Tao Sun, Dustin Armstrong, Scott Borneman, Chunyu Yang, Stephanie Austin, Priya S. Kishnani, and Baodong Sun. “Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.” J Mol Med (Berl) 95, no. 5 (May 2017): 513–21. https://doi.org/10.1007/s00109-017-1505-9.Full Text Open Access Copy Link to Item
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Yi, Haiqing, Quan Zhang, Elizabeth D. Brooks, Chunyu Yang, Beth L. Thurberg, Priya S. Kishnani, and Baodong Sun. “Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy.” Hum Gene Ther 28, no. 3 (March 2017): 286–94. https://doi.org/10.1089/hum.2016.099.Full Text Open Access Copy Link to Item
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Austin, Stephanie L., Andrew Chiou, Baodong Sun, Laura E. Case, Kenny Govendrageloo, Perrin Hansen, and Priya S. Kishnani. “Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for a patient presenting with a PRKAG2 mutation.” Mol Genet Metab 120, no. 1–2 (2017): 96–100. https://doi.org/10.1016/j.ymgme.2016.09.006.Full Text Open Access Copy Link to Item
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Yi, Haiqing, Fengqin Gao, Stephanie Austin, Priya S. Kishnani, and Baodong Sun. “Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV.” Mol Genet Metab Rep 9 (December 2016): 31–33. https://doi.org/10.1016/j.ymgmr.2016.09.008.Full Text Open Access Copy Link to Item
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Sun, Tao, Haiqing Yi, Chunyu Yang, Priya S. Kishnani, and Baodong Sun. “Starch Binding Domain-containing Protein 1 Plays a Dominant Role in Glycogen Transport to Lysosomes in Liver.” J Biol Chem 291, no. 32 (August 5, 2016): 16479–84. https://doi.org/10.1074/jbc.C116.741397.Full Text Open Access Copy Link to Item
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Brooks, Elizabeth D., Haiqing Yi, Stephanie L. Austin, Beth L. Thurberg, Sarah P. Young, John C. Fyfe, Priya S. Kishnani, and Baodong Sun. “Natural Progression of Canine Glycogen Storage Disease Type IIIa.” Comp Med 66, no. 1 (February 2016): 41–51.Open Access Copy Link to Item
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Yi, Haiqing, Quan Zhang, Chunyu Yang, Priya S. Kishnani, and Baodong Sun. “A Modified Enzymatic Method for Measurement of Glycogen Content in Glycogen Storage Disease Type IV.” Jimd Rep 30 (2016): 89–94. https://doi.org/10.1007/8904_2015_522.Full Text Open Access Copy Link to Item
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Sun, Baodong, Elizabeth D. Brooks, and Dwight D. Koeberl. “Preclinical Development of New Therapy for Glycogen Storage Diseases.” Curr Gene Ther 15, no. 4 (2015): 338–47. https://doi.org/10.2174/1566523215666150630132253.Full Text Open Access Copy Link to Item
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Yi, Haiqing, Elizabeth D. Brooks, Beth L. Thurberg, John C. Fyfe, Priya S. Kishnani, and Baodong Sun. “Correction of glycogen storage disease type III with rapamycin in a canine model.” J Mol Med (Berl) 92, no. 6 (June 2014): 641–50. https://doi.org/10.1007/s00109-014-1127-4.Full Text Open Access Copy Link to Item
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Sun, Baodong, Suhrad G. Banugaria, Sean N. Prater, Trusha T. Patel, Keri Fredrickson, Douglas J. Ringler, Antonin de Fougerolles, Amy S. Rosenberg, Herman Waldmann, and Priya S. Kishnani. “Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.” Mol Genet Metab Rep 1 (2014): 446–50. https://doi.org/10.1016/j.ymgmr.2014.08.005.Full Text Open Access Copy Link to Item
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Yi, Haiqing, Keri B. Fredrickson, Stuti Das, Priya S. Kishnani, and Baodong Sun. “Stbd1 is highly elevated in skeletal muscle of Pompe disease mice but suppression of its expression does not affect lysosomal glycogen accumulation.” Mol Genet Metab 109, no. 3 (July 2013): 312–14. https://doi.org/10.1016/j.ymgme.2013.05.004.Full Text Open Access Copy Link to Item
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Brooks, Elizabeth Drake, Dianne Little, Ramamani Arumugam, Baodong Sun, Sarah Curtis, Amanda Demaster, Michael Maranzano, et al. “Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine Glycogen Storage Disease type Ia.” Mol Genet Metab 109, no. 2 (June 2013): 161–70. https://doi.org/10.1016/j.ymgme.2013.03.018.Full Text Open Access Copy Link to Item
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Sun, Baodong, Keri Fredrickson, Stephanie Austin, Adviye A. Tolun, Beth L. Thurberg, William E. Kraus, Deeksha Bali, Yuan-Tsong Chen, and Priya S. Kishnani. “Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for glycogen storage disease type III.” Mol Genet Metab 108, no. 2 (February 2013): 145–47. https://doi.org/10.1016/j.ymgme.2012.12.002.Full Text Open Access Copy Link to Item
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Li, Songtao, Baodong Sun, Mats I. Nilsson, Andrew Bird, Mark A. Tarnopolsky, Beth L. Thurberg, Deeksha Bali, and Dwight D. Koeberl. “Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease.” Faseb J 27, no. 1 (January 2013): 34–44. https://doi.org/10.1096/fj.12-207472.Full Text Open Access Copy Link to Item
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Yi, Haiqing, Beth L. Thurberg, Sarah Curtis, Stephanie Austin, John Fyfe, Dwight D. Koeberl, Priya S. Kishnani, and Baodong Sun. “Characterization of a canine model of glycogen storage disease type IIIa.” Dis Model Mech 5, no. 6 (November 2012): 804–11. https://doi.org/10.1242/dmm.009712.Full Text Open Access Copy Link to Item
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Zhang, Ping, Baodong Sun, Takuya Osada, Ramona Rodriguiz, Xiao Yi Yang, Xiaoyan Luo, Alex R. Kemper, Timothy M. Clay, and Dwight D. Koeberl. “Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease.” Hum Gene Ther 23, no. 5 (May 2012): 460–72. https://doi.org/10.1089/hum.2011.063.Full Text Open Access Copy Link to Item
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Koeberl, Dwight D., Xiaoyan Luo, Baodong Sun, Alison McVie-Wylie, Jian Dai, Songtao Li, Suhrad G. Banugaria, Y. -. T. Chen, and Deeksha S. Bali. “Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle.” Mol Genet Metab 103, no. 2 (June 2011): 107–12. https://doi.org/10.1016/j.ymgme.2011.02.006.Full Text Open Access Copy Link to Item
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Sun, B., S. Li, A. Bird, and D. D. Koeberl. “Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease.” Gene Ther 17, no. 12 (December 2010): 1500–1505. https://doi.org/10.1038/gt.2010.109.Full Text Open Access Copy Link to Item
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Sun, Baodong, Songtao Li, Andrew Bird, Haiqing Yi, Alex Kemper, Beth L. Thurberg, and Dwight D. Koeberl. “Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.” J Gene Med 12, no. 11 (November 2010): 881–91. https://doi.org/10.1002/jgm.1511.Full Text Open Access Copy Link to Item
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Sun, Baodong, Michael D. Kulis, Sarah P. Young, Amy C. Hobeika, Songtao Li, Andrew Bird, Haoyue Zhang, et al. “Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.” Mol Ther 18, no. 2 (February 2010): 353–60. https://doi.org/10.1038/mt.2009.195.Full Text Link to Item
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Sun, Baodong, Haoyue Zhang, Andrew Bird, Songtao Li, Sarah P. Young, and Dwight D. Koeberl. “Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression.” J Gene Med 11, no. 10 (October 2009): 913–20. https://doi.org/10.1002/jgm.1372.Full Text Link to Item
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Sun, Baodong, Songtao Li, Liu Yang, Tirupapuliyur Damodaran, Dev Desai, Anna Mae Diehl, Oscar Alzate, and Dwight D. Koeberl. “Activation of glycolysis and apoptosis in glycogen storage disease type Ia.” Mol Genet Metab 97, no. 4 (August 2009): 267–71. https://doi.org/10.1016/j.ymgme.2009.04.003.Full Text Link to Item
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Sun, Baodong, Sarah P. Young, Ping Li, Chunhui Di, Talmage Brown, Maja Z. Salva, Songtao Li, et al. “Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy.” Mol Ther 16, no. 8 (August 2008): 1366–71. https://doi.org/10.1038/mt.2008.133.Full Text Link to Item
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Koeberl, Dwight D., Carlos Pinto, Baodong Sun, Songtao Li, Daniel M. Kozink, Daniel K. Benjamin, Amanda K. Demaster, et al. “AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia.” Mol Ther 16, no. 4 (April 2008): 665–72. https://doi.org/10.1038/mt.2008.15.Full Text Link to Item
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Koeberl, Dwight D., Baodong Sun, Andrew Bird, Yuan-Tsong Chen, Kazuhiro Oka, and Lawrence Chan. “Corrigendum to "Efficacy of Helper-dependent Adenovirus Vector-mediated Gene Therapy in Murine Glycogen Storage Disease Type Ia".” Mol Ther 15, no. 11 (November 2007): 2051. https://doi.org/10.1038/sj.mt.6300269.Full Text Link to Item
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Sun, Baodong, Andrew Bird, Sarah P. Young, Priya S. Kishnani, Y. -. T. Chen, and Dwight D. Koeberl. “Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.” Am J Hum Genet 81, no. 5 (November 2007): 1042–49. https://doi.org/10.1086/522236.Full Text Link to Item
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Koeberl, Dwight D., B. Sun, A. Bird, Y. T. Chen, K. Oka, and L. Chan. “Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia.” Mol Ther 15, no. 7 (July 2007): 1253–58. https://doi.org/10.1038/sj.mt.6300188.Full Text Link to Item
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Pinto, C. R., T. R. Brown, V. Vaughn, A. Bird, B. D. Sun, and D. D. Koeberl. “Efficacy of gene therapy in canine glycogen storage disease type IA.” Journal of Veterinary Internal Medicine 21, no. 3 (May 1, 2007): 606–606.Link to Item
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Koeberl, D. D., B. D. Sun, T. V. Damodaran, T. Brown, D. S. Millington, J. K. Benjamin, A. Bird, et al. “Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia (Gene Therapy (2006) vol. 13 (1281-1289) 10.1038/sj.gt.3302774).” Gene Therapy 14, no. 3 (February 1, 2007): 281. https://doi.org/10.1038/sj.gt.3302903.Full Text
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Sun, Baodong, Haoyue Zhang, Daniel K. Benjamin, Talmage Brown, Andrew Bird, Sarah P. Young, Alison McVie-Wylie, Y. -. T. Chen, and Dwight D. Koeberl. “Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.” Mol Ther 14, no. 6 (December 2006): 822–30. https://doi.org/10.1016/j.ymthe.2006.08.001.Full Text Link to Item
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Koeberl, D. D., B. D. Sun, T. V. Damodaran, T. Brown, D. S. Millington, D. K. Benjamin, A. Bird, et al. “Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia (Gene Therapy (2006) vol. 13 (1281-1289) 10.1038/sj.gt.3302774)).” Gene Therapy 13, no. 19 (October 1, 2006): 1430. https://doi.org/10.1038/sj.gt.3302857.Full Text
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Koeberl, D. D., B. D. Sun, T. V. Damodaran, T. Brown, D. S. Millington, D. K. Benjamin, A. Bird, et al. “Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.” Gene Ther 13, no. 17 (September 2006): 1281–89. https://doi.org/10.1038/sj.gt.3302774.Full Text Link to Item
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Franco, Luis M., Baodong Sun, Xiaoyi Yang, Andrew Bird, Haoyue Zhang, Ayn Schneider, Talmage Brown, et al. “Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.” Mol Ther 12, no. 5 (November 2005): 876–84. https://doi.org/10.1016/j.ymthe.2005.04.024.Full Text Link to Item
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Sun, Baodong, Haoyue Zhang, Luis M. Franco, Talmage Brown, Andrew Bird, Ayn Schneider, and Dwight D. Koeberl. “Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.” Mol Ther 11, no. 6 (June 2005): 889–98. https://doi.org/10.1016/j.ymthe.2005.01.012.Full Text Link to Item
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Sun, Baodong, Haoyue Zhang, Luis M. Franco, Sarah P. Young, Ayn Schneider, Andrew Bird, Andrea Amalfitano, Y. -. T. Chen, and Dwight D. Koeberl. “Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.” Mol Ther 11, no. 1 (January 2005): 57–65. https://doi.org/10.1016/j.ymthe.2004.10.004.Full Text Link to Item
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Sun, Baodong, Y. -. T. Chen, Andrew Bird, Fang Xu, Yang-Xun Hou, Andrea Amalfitano, and Dwight D. Koeberl. “Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector.” Mol Ther 7, no. 4 (April 2003): 467–77. https://doi.org/10.1016/s1525-0016(03)00022-4.Full Text Link to Item
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Sun, Bao-dong, Y. -. T. Chen, Andrew Bird, Andrea Amalfitano, and Dwight D. Koeberl. “Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.” Mol Ther 7, no. 2 (February 2003): 193–201. https://doi.org/10.1016/s1525-0016(02)00055-2.Full Text Link to Item
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Plonowski, Artur, Andrew V. Schally, Attila Nagy, Baodong Sun, and Gabor Halmos. “Effective treatment of experimental DU-145 prostate cancers with targeted cytotoxic somatostatin analog AN-238.” Int J Oncol 20, no. 2 (February 2002): 397–402.Link to Item
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Szepeshazi, Karoly, Andrew V. Schally, Gabor Halmos, Patricia Armatis, Francine Hebert, Baodong Sun, Anita Feil, Hippokratis Kiaris, and Attila Nagy. “Targeted cytotoxic somatostatin analogue AN-238 inhibits somatostatin receptor-positive experimental colon cancers independently of their p53 status.” Cancer Res 62, no. 3 (February 1, 2002): 781–88.Link to Item
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Ehrlich, M., K. L. Buchanan, F. Tsien, G. Jiang, B. Sun, W. Uicker, C. M. Weemaes, et al. “DNA methyltransferase 3B mutations linked to the ICF syndrome cause dysregulation of lymphogenesis genes.” Hum Mol Genet 10, no. 25 (December 1, 2001): 2917–31. https://doi.org/10.1093/hmg/10.25.2917.Full Text Link to Item
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Tsien, F., B. Sun, N. E. Hopkins, V. Vedanarayanan, D. Figlewicz, S. Winokur, and M. Ehrlich. “Methylation of the FSHD syndrome-linked subtelomeric repeat in normal and FSHD cell cultures and tissues.” Mol Genet Metab 74, no. 3 (November 2001): 322–31. https://doi.org/10.1006/mgme.2001.3219.Full Text Link to Item
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Sun, B., G. Jiang, M. A. Zaydan, V. F. La Russa, H. Safah, and M. Ehrlich. “ABL1 promoter methylation can exist independently of BCR-ABL transcription in chronic myeloid leukemia hematopoietic progenitors.” Cancer Res 61, no. 18 (September 15, 2001): 6931–37.Link to Item
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Szepeshazi, K., A. V. Schally, G. Halmos, B. Sun, F. Hebert, B. Csernus, and A. Nagy. “Targeting of cytotoxic somatostatin analog AN-238 to somatostatin receptor subtypes 5 and/or 3 in experimental pancreatic cancers.” Clin Cancer Res 7, no. 9 (September 2001): 2854–61.Link to Item
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Plonowski, A., A. Nagy, A. V. Schally, B. Sun, K. Groot, and G. Halmos. “In vivo inhibition of PC-3 human androgen-independent prostate cancer by a targeted cytotoxic bombesin analogue, AN-215.” Int J Cancer 88, no. 4 (November 15, 2000): 652–57. https://doi.org/10.1002/1097-0215(20001115)88:4<652::aid-ijc21>3.0.co;2-1.Full Text Link to Item
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Chatzistamou, I., A. V. Schally, B. Sun, P. Armatis, and K. Szepeshazi. “Inhibition of growth of OV-1063 human epithelial ovarian cancers and c- jun and c- fos oncogene expression by bombesin antagonists.” Br J Cancer 83, no. 7 (October 2000): 906–13. https://doi.org/10.1054/bjoc.2000.1374.Full Text Link to Item
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Halmos, G., B. Sun, A. V. Schally, F. Hebert, and A. Nagy. “Human ovarian cancers express somatostatin receptors.” J Clin Endocrinol Metab 85, no. 10 (October 2000): 3509–12. https://doi.org/10.1210/jcem.85.10.3509.Full Text Link to Item
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Halmos, G., A. V. Schally, B. Sun, R. Davis, D. G. Bostwick, and A. Plonowski. “High expression of somatostatin receptors and messenger ribonucleic acid for its receptor subtypes in organ-confined and locally advanced human prostate cancers.” J Clin Endocrinol Metab 85, no. 7 (July 2000): 2564–71. https://doi.org/10.1210/jcem.85.7.6698.Full Text Link to Item
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Sun, B., A. V. Schally, and G. Halmos. “The presence of receptors for bombesin/GRP and mRNA for three receptor subtypes in human ovarian epithelial cancers.” Regul Pept 90, no. 1–3 (June 30, 2000): 77–84. https://doi.org/10.1016/s0167-0115(00)00114-2.Full Text Link to Item
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Kahán, Z., B. Sun, A. V. Schally, J. M. Arencibia, R. Z. Cai, K. Groot, and G. Halmos. “Inhibition of growth of MDA-MB-468 estrogen-independent human breast carcinoma by bombesin/gastrin-releasing peptide antagonists RC-3095 and RC-3940-II.” Cancer 88, no. 6 (March 15, 2000): 1384–92. https://doi.org/10.1002/(sici)1097-0142(20000315)88:6<1384::aid-cncr16>3.0.co;2-q.Full Text Link to Item
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Sun, B., G. Halmos, A. V. Schally, X. Wang, and M. Martinez. “Presence of receptors for bombesin/gastrin-releasing peptide and mRNA for three receptor subtypes in human prostate cancers.” Prostate 42, no. 4 (March 1, 2000): 295–303. https://doi.org/10.1002/(sici)1097-0045(20000301)42:4<295::aid-pros7>3.0.co;2-b.Full Text Link to Item
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Kiaris, H., A. V. Schally, A. Nagy, B. Sun, K. Szepeshazi, and G. Halmos. “Regression of U-87 MG human glioblastomas in nude mice after treatment with a cytotoxic somatostatin analog AN-238.” Clin Cancer Res 6, no. 2 (February 2000): 709–17.Link to Item
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Kiaris, H., A. V. Schally, B. Sun, P. Armatis, and K. Groot. “Inhibition of growth of human malignant glioblastoma in nude mice by antagonists of bombesin/gastrin-releasing peptide.” Oncogene 18, no. 50 (November 25, 1999): 7168–73. https://doi.org/10.1038/sj.onc.1203213.Full Text Link to Item
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Kiaris, H., A. V. Schally, A. Nagy, B. Sun, P. Armatis, and K. Szepeshazi. “Targeted cytotoxic analogue of bombesin/gastrin-releasing peptide inhibits the growth of H-69 human small-cell lung carcinoma in nude mice.” Br J Cancer 81, no. 6 (November 1999): 966–71. https://doi.org/10.1038/sj.bjc.6690794.Full Text Link to Item
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Kahán, Z., A. Nagy, A. V. Schally, F. Hebert, B. Sun, K. Groot, and G. Halmos. “Inhibition of growth of MX-1, MCF-7-MIII and MDA-MB-231 human breast cancer xenografts after administration of a targeted cytotoxic analog of somatostatin, AN-238.” Int J Cancer 82, no. 4 (August 12, 1999): 592–98. https://doi.org/10.1002/(sici)1097-0215(19990812)82:4<592::aid-ijc20>3.0.co;2-0.Full Text Link to Item
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Plonowski, A., A. V. Schally, A. Nagy, B. Sun, and K. Szepeshazi. “Inhibition of PC-3 human androgen-independent prostate cancer and its metastases by cytotoxic somatostatin analogue AN-238.” Cancer Res 59, no. 8 (April 15, 1999): 1947–53.Link to Item
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Conference Papers
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Gibson, Rebecca A., Jeong-A Lim, Su Jin Choi, Deeksha Bali, Sarah Young, Rebecca Koch, Baodong Sun, Aravind Asokan, and Priya S. Kishnani. “SEVERE PROGRESSION OF LIVER DISEASE IN AN AGING PHKG2(-/-) MOUSE MODEL RECAPITULATES GSD IX G2 PATIENT PHENOTYPE.” In Molecular Genetics and Metabolism, 135:272–272, 2022.Link to Item
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Gibson, Rebecca A., Jeong-A Lim, Su Jin Choi, Rebecca L. Koch, Deeksha Bali, Sarah Young, William Jeck, Baodong Sun, Aravind Asokan, and Priya S. Kishnani. “Liver Directed AAV Gene Therapy Reverses Progression of Glycogen Storage Disease Type IX gamma 2 in Mice.” In Molecular Therapy, 30:478–79, 2022.Link to Item
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Lim, Jeong-A, Su Jin Choi, Priya S. Kishnani, and Baodong Sun. “Long-Term Correction of Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using a Dual Promoter to Express Bacterial Pullulanase.” In Molecular Therapy, 30:119–20, 2022.Link to Item
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Yi, Haiqing, Jeong-A Lim, Priya S. Kishnani, and Baodong Sun. “AAV Gene Therapy Effectively Transduces the Central Nervous System and Prevents Polyglucosan Body Formation in Adult Polyglucosan Body Disease Mice.” In Molecular Therapy, 30:467–68, 2022.Link to Item
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Choi, Su Jin, John Yi, Thomas F. Tedder, Dwight D. Koeberl, Priya S. Kishnani, and Baodong Sun. “Immunosuppression with bortezomib and anti-CD20 mAb is effective in reducing neutralizing antibodies to allow repeated AAV administration in mice.” In Molecular Genetics and Metabolism, 132:S25–26. Elsevier BV, 2021. https://doi.org/10.1016/j.ymgme.2020.12.041.Full Text
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Choi, Su Jin, John Yi, Thomas F. Tedder, Dwight D. Koeberl, Priya S. Kishnani, and Baodong Sun. “Depletion of Pre-Existing Anti-AAV Neutralizing Antibodies by a Combination Immunosuppressive Treatment with Bortezomib and CD20 mAb Allows Successful Vector Re-Administration in Mice.” In Molecular Therapy, 29:361–361, 2021.Link to Item
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Gibson, Rebecca, Jeong-A Lim, Su Jin Choi, Leticia Flores, Lani Clinton, Deeksha Bali, Sarah Young, Aravind Asokan, Baodong Sun, and Priya Kishnani. “Characterization of Liver Pathology in a Novel GSD IX y2 Mouse Model.” In Molecular Therapy, 29:239–239, 2021.Link to Item
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Lim, Jeong-A, Priya S. Kishnani, and Baodong Sun. “AAV-Mediated Gene Therapy Using a Novel Dual Promoter Prevents Pullulanase-Induced Cytotoxic T Lymphocyte Response and Corrects Major Affected Tissues in GSD IIIa Mice.” In Molecular Therapy, 29:235–36, 2021.Link to Item
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Choi, Su Jin, John Yi, Thomas F. Tedder, Dwight D. Koeberl, Priya S. Kishnani, and Baodong Sun. “A Combination Immunosuppression Treatment with Bortezomib and an Anti-CD20 mAb Enables Successful Re-Administration of AAV8 Vector in Mice.” In Molecular Therapy, 28:521–521, 2020.Link to Item
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Lim, Jeong-A, Su Jin Choi, Fengqin Gao, Priya S. Kishnani, and Baodong Sun. “AAV-Mediated Gene Therapy for Glycogen Storage Disease Type III Using a Bacterial Glycogen Debranching Enzyme.” In Molecular Therapy, 28:307–8, 2020.Link to Item
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Yi, Haiqing, Tao Sun, Fengqin Gao, Priya S. Kishnani, and Baodong Sun. “Transgene Immunogenicity Has Significant Effects on Gene Therapy in a Mouse Model of Adult Polyglucosan Body Disease.” In Molecular Therapy, 28:263–263, 2020.Link to Item
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Brooks, Elizabeth D., Haiqing Yi, Baodong Sun, Jeong-A Lim, Carine A. Halaby, Lani K. Clinton, Alisha M. Mavis, et al. “COMPARATIVE LIVER PATHOLOGY IN GLYCOGEN STORAGE DISEASE TYPE IIIA.” In Molecular Genetics and Metabolism, 127:256–57. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2019.Link to Item
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Lim, Jeong-A, Su Jin Choi, Fengqin Gao, Priya Kishnani, and Baodong Sun. “Correction of Glycogen Storage Disease Type III with an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme.” In Molecular Therapy, 27:17–17. CELL PRESS, 2019.Link to Item
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Brooks, Elizabeth D., Haiqing Yi, Baodong Sun, Jeong-A Lim, Carine A. Halaby, Lani K. Clinton, Alisha M. Mavis, et al. “COMPARATIVE LIVER PATHOLOGY IN GLYCOGEN STORAGE DISEASE TYPE IIIA.” In Molecular Genetics and Metabolism, 126:291–291. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2019.Link to Item
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Lim, Jeong-A, Haiqing Yi, Fengqin Gao, Priya Kishinani, and Baodong Sun. “A Single Intravenous Injection of an AAV-PHP. B Vector Encoding Human Acid alpha-Glucosidase Corrects Both Muscle and Brain Defects in Murine Pompe Disease.” In Molecular Therapy, 26:382–83. CELL PRESS, 2018.Link to Item
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Lim, Han-Hyuk, Haiqing Yi, Takashi K. Kishimoto, Fengqin Gao, Baodong Sun, and Priya S. Kishnani. “Immunomodulation to enzyme replacement therapy with tolerogenic nanoparticles containing rapamycin in a murine model of Pompe disease.” In Molecular Genetics and Metabolism, 120:S83–84. Elsevier BV, 2017. https://doi.org/10.1016/j.ymgme.2016.11.202.Full Text
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Sun, Baodong, Tao Sun, Dustin Armstrong, Haiqing Yi, Chunyu Yang, Stephanie Austin, and Priya S. Kishnani. “New perspectives for ERT in Pompe disease: Extending the action of the enzyme to cytosolic targets.” In Molecular Genetics and Metabolism, 117:S110–11. Elsevier BV, 2016. https://doi.org/10.1016/j.ymgme.2015.12.453.Full Text
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Brooks, Elizabeth J., Dianne Little, Ramamani Arumugam, Baodong Sun, Sarah Curtis, Amanda DeMaster, Michael Maranzano, et al. “Pathogenesis of Growth Failure in Murine and Canine Glycogen Storage Disease-Ia and Partial Reversal of Growth Failure Using Adeno-Associated Viral Vector Therapy.” In Molecular Therapy, 21:S105–6. NATURE PUBLISHING GROUP, 2013.Link to Item
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Austin, S. L., A. D. Proia, R. Kanter, E. Drake, B. Sun, S. B. Wechsler, and P. S. Kishnani. “CARDIAC PATHOLOGY AND CLINICAL FINDINGS COMPARISON IN PATIENTS AND DOGS WITH GLYCOGEN STORAGE DISEASE TYPE III.” In Journal of Inherited Metabolic Disease, 35:S81–S81. SPRINGER, 2012.Link to Item
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Sun, B., H. Yi, B. Thurberg, S. Austin, J. Fyfe, D. Koeberl, and P. Kishnani. “CHARACTERIZATION OF LIVER AND MUSCLE PHENOTYPES IN A CANINE MODEL OF GLYCOGEN STORAGE DISEASE TYPE IIIA.” In Journal of Inherited Metabolic Disease, 35:S80–S80. SPRINGER, 2012.Link to Item
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Sun, Baodong, Haiqing Yi, Beth Thurberg, Sarah Curtis, Stephanie Austin, John Fyfe, Dwight Koeberl, and Priya Kishnani. “A canine model of glycogen storage disease type III.” In Molecular Genetics and Metabolism, 105:355–56. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2012.Link to Item
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Yi, Haiqing, Baodong Sun, Keri Fredrickson, Elizabeth Drake, Beth Thurberg, John Fyfe, Karen Corneliussen, Deeksha Bali, and Priya Kishnani. “Rapamycin is a potential therapy for glycogen storage disease type III.” In Molecular Genetics and Metabolism, 105:364–364. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2012.Link to Item
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Koeberl, Dwight, Baodong Sun, Alison McVie-Wylie, Jian Dai, Songtao Li, Y. -. T. Chen, and Deeksha Bali. “Mannose-6-Phosphate receptor expression modulates efficacy from enzyme replacement therapy in Pompe disease.” In Molecular Genetics and Metabolism, 102:S25–S25. Elsevier BV, 2011. https://doi.org/10.1016/j.ymgme.2010.11.085.Full Text
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Sun, Baodong, Michael Kulis, Songtao Li, Yifan Li, Wesley Burks, Priya S. Kishnani, and Dwight D. Koeberl. “Desensitization through Immunomodulatory Gene Therapy Suppresses the Antibody Response to Enzyme Replacement Therapy in Murine Pompe Disease.” In Molecular Therapy, 17:S344–S344. NATURE PUBLISHING GROUP, 2009.Link to Item
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Sun, Baodong, Stephen Hauschka, Andrew Bird, Richard Auten, and Divight Koeberl. “Muscle-targeted gene therapy in glycogen storage disease type II with adeno-associated virus serotypes 7, 8, and 9.” In Molecular Genetics and Metabolism, 93:S38–39. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2008.Link to Item
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Koeberl, Dwight D., Baodong Sun, Andrew Bird, Sarah P. Young, Y. -. T. Chen, and Priya S. Kishnani. “70 Enhanced response to enzyme replacement therapy in Pompe disease following the induction of immune tolerance.” In Molecular Genetics and Metabolism, 92:27–27. Elsevier BV, 2007. https://doi.org/10.1016/j.ymgme.2007.08.075.Full Text
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Koeberl, D. D., B. Sun, C. Pinto, T. Brown, A. Bird, and Y. T. Chen. “Highly efficacious gene therapy in glycogen storage disease type Ia (GSD-Ia) with a double-stranded AAV vector.” In Molecular Genetics and Metabolism, 90:255–56. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2007.Link to Item
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Koeberl, Dwight D., Lopamudra Banerjee, Andrew Bird, Baodong Sun, and Y. T. Chen. “487. Liver-Targeted Gene Therapy in Glycogen Storage Disease Type Ia (GSD-Ia) Requires Widespread, Regulated Glucose-6-phosphatase.” In Molecular Therapy, 13:S189–S189. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.557.Full Text
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Koeberl, Dwight D., Lopamudra Banerjee, Baodong Sun, Andrew Bird, Y. T. Chen, Kazuhiro Hiro, and Lawrence Chan. “211. Development of Helper-Dependent Adenovirus (HDAd) Vectors for Gene Therapy in the Mouse Model for Glycogen Storage Disease Type Ia (GSD-Ia).” In Molecular Therapy, 13:S81–82. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.236.Full Text
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Sun, Baodong, Ping Li, Brown Talmage, Bird Andrew, Zhen Yan, and Dwight D. Koeberl. “563. Correction of Glycogen Storage Disease Type II by Systemic Delivery of an AAV2/8 Vector Containing a Muscle-Specific Promoter.” In Molecular Therapy, 13:S217–S217. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.636.Full Text
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Koeberl, D. D., B. D. Sun, L. Franco, A. Schneider, A. Bird, E. Faulkner, and Y. T. Chen. “Efficacy of an adeno-associated virus (AAV) vector administered to glycogen storage disease type Ia (GSD-Ia) mice at mid-infancy.” In Molecular Genetics and Metabolism, 84:226–27. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2005.Link to Item
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Koeberl, D. D., B. D. Sun, L. Franco, A. Bird, E. Faulkner, and Y. T. Chen. “Development of adeno-associated virus (AAV) vectors for gene therapy in the mouse model for glycogen storage disease type Ia (GSD-Ia).” In Molecular Therapy, 9:S404–S404. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2004.Link to Item
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Zhang, H. Y., B. D. Sun, L. Franco, A. Bird, A. Schneider, and D. D. Koeberl. “Correction of glycogen storage disease type II by intramuscular administration of an adeno-associated virus 6 (AAV2/6) vector containing a muscle-specific promoter.” In Molecular Therapy, 9:S329–30. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2004.Link to Item
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Franco, Luis M., Baodong Sun, Andrew Bird, Haoyue Zhang, Andrea Amalfitano, Y. -. T. Chen, and Dwight D. Koeberl. “339. Sustained, High-Level Expression of Human Acid Alpha-Glucosidase and Correction of Glycogen Storage Disease Type II (GSD II) with an Adeno-Associated Virus 8 (AAV2/8) Vector Containing a Liver-Specific Promoter.” In Molecular Therapy, 9:S129–S129. Elsevier BV, 2004. https://doi.org/10.1038/mt.2004.1400.Full Text
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Sun, B. D., Y. T. Chen, A. Bird, F. Xu, A. Amalfitano, and D. D. Koeberl. “Correction of glycogen storage disease type II (GSD II) by intramuscular administration of an adeno-associated virus (AAV) vector pseudotyped as AAV6.” In Molecular Therapy, 7:S391–S391. ACADEMIC PRESS INC ELSEVIER SCIENCE, 2003.Link to Item
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Koeberl, D. D., B. D. Sun, B. Andrew, A. Amalfitano, and Y. T. Chen. “Development of a hybrid adenovirus/adeno-associated virus vector for gene therapy in glycogen storage disease type II.” In Pediatric Research, 53:259A-260A. INT PEDIATRIC RESEARCH FOUNDATION, INC, 2003.Link to Item
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