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Human Vaccines & Immunotherapeutics Volume 8, 2012 - Issue 10
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Commentary

Teaching tolerance

New approaches to enzyme replacement therapy for Pompe disease

Leslie P. Cousens EpiVax, Inc.; Providence, RI USA
,
Federico Mingozzi Children’s Hospital of Philadelphia; Philadelphia, PA USA
,
Sander van der Marel AMT/UniQure; Amsterdam, Netherlands; Department of Gastroenterology & Hepatology; LUMC; Leiden, Netherlands
,
Yan Su Uniformed Services University of Health Services (USUHS); Bethesda, MD USA
,
Richard Garman EpiVax, Inc.; Providence, RI USA
,
Valerie Ferreira AMT/UniQure; Amsterdam, Netherlands
,
William Martin EpiVax, Inc.; Providence, RI USA
,
David W. Scott Uniformed Services University of Health Services (USUHS); Bethesda, MD USA
&
Anne S De Groot EpiVax, Inc.; Providence, RI USA; Institute for Immunology and Informatics; University of Rhode Island; Providence, RI USACorrespondence[email protected]
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Pages 1459-1464 | Received 29 Jun 2012, Accepted 07 Jul 2012, Published online: 01 Oct 2012

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Anne S De Groot. (2013) Do Tregitopes have the potential to impact the current treatment landscape of autoimmune diseases?. Expert Review of Clinical Immunology 9:12, pages 1155-1157.
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Giancarlo Parenti & Generoso Andria. (2013) Pharmacotherapy of Pompe disease. Expert Opinion on Orphan Drugs 1:6, pages 457-471.
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Leiling Liu, Jiahui Hu, Hao Lei, Huali Qin, Chunfang Wang, Yajun Gui & Danyan Xu. (2023) Regulatory T Cells in Pathological Cardiac Hypertrophy: Mechanisms and Therapeutic Potential. Cardiovascular Drugs and Therapy.
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Line Gutte Borgwardt, Ferdinando Ceravolo, Giulia Zardi, Andrea Ballabeni & Allan Meldgaard Lund. (2022) Relationship between MAN2B1 genotype/subcellular localization subgroups, antidrug antibody detection, and long‐term velmanase alfa treatment outcomes in patients with alpha‐mannosidosis . JIMD Reports 64:2, pages 187-198.
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Jia-Xin Wan, Gyutae Lim, Jinhyuk Lee, Xiao-Bao Sun, De-Ying Gao, Yue-Xiu Si, Xin-Lei Shi, Guo-Ying Qian, Qian Wang & Yong-Doo Park. (2019) Inhibitory effect of phloroglucinol on α-glucosidase: Kinetics and molecular dynamics simulation integration study. International Journal of Biological Macromolecules 124, pages 771-779.
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J. Hordeaux, L. Dubreil, C. Robveille, J. Deniaud, Q. Pascal, B. Dequéant, J. Pailloux, L. Lagalice, M. Ledevin, C. Babarit, P. Costiou, F. Jamme, M. Fusellier, Y. Mallem, C. Ciron, C. Huchet, C. Caillaud & M-A Colle. (2017) Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease. Acta Neuropathologica Communications 5:1.
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Han-Hyuk Lim, Haiqing Yi, Takashi K. Kishimoto, Fengqin Gao, Baodong Sun & Priya S. Kishnani. (2017) A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease. Molecular Genetics and Metabolism Reports 13, pages 18-22.
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Moanaro Biswas, Geoffrey L. Rogers, Alexandra Sherman, Barry J. Byrne, David M. Markusic, Haiyan Jiang & Roland W. Herzog. (2017) Combination therapy for inhibitor reversal in haemophilia A using monoclonal anti-CD20 and rapamycin. Thrombosis and Haemostasis 117:01, pages 33-43.
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Boris Gorovits, Adrienne Clements-Egan, Mary Birchler, Meina Liang, Heather Myler, Kun Peng, Shobha Purushothama, Manoj Rajadhyaksha, Laura Salazar-Fontana, Crystal Sung & Li Xue. (2016) Pre-existing Antibody: Biotherapeutic Modality-Based Review. The AAPS Journal 18:2, pages 311-320.
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Paul Harmatz. (2015) Enzyme Replacement Therapies and Immunogenicity in Lysosomal Storage Diseases: Is There a Pattern?. Clinical Therapeutics 37:9, pages 2130-2134.
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Leslie P. Cousens, Leonard Moise & Anne S. De Groot. 2015. Biobetters. Biobetters 63 77 .
Xuan Li, Zhi-Rong Lü, Dong Shen, Yi Zhan, Jun-Mo Yang, Yong-Doo Park, Hai-Meng Zhou, Qing Sheng & Jinhyuk Lee. (2014) The inhibitory role of Co2+ on α-glucosidase: Inhibition kinetics and molecular dynamics simulation integration study. Process Biochemistry 49:11, pages 1913-1919.
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Sushrusha Nayak, Phillip A. Doerfler, Stacy L. Porvasnik, Denise D. Cloutier, Richie Khanna, Ken J. Valenzano, Roland W. Herzog & Barry J. Byrne. (2014) Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent. PLoS ONE 9:6, pages e98336.
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Daniel J Hui, Etiena Basner-Tschakarjan, Yifeng Chen, Robert J Davidson, George Buchlis, Mustafa Yazicioglu, Gary C Pien, Jonathan D Finn, Virginia Haurigot, Alex Tai, David W Scott, Leslie P Cousens, Shangzhen Zhou, Anne S De Groot & Federico Mingozzi. (2013) Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes. Molecular Therapy 21:9, pages 1727-1737.
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Yan Su, Robert Rossi, Anne S De Groot & David W Scott. (2013) Regulatory T cell epitopes (Tregitopes) in IgG induce tolerance in vivo and lack immunogenicity per se. Journal of Leukocyte Biology 94:2, pages 377-383.
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Leslie P. Cousens, Yan Su, Elizabeth McClaine, Xin Li, Frances Terry, Robert Smith, Jinhee Lee, William Martin, David W. Scott & Anne S. De Groot. (2013) Application of IgG-Derived Natural Treg Epitopes (IgG Tregitopes) to Antigen-Specific Tolerance Induction in a Murine Model of Type 1 Diabetes. Journal of Diabetes Research 2013, pages 1-17.
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