دورية أكاديمية
Innovative approach in Pompe disease therapy: Induction of immune tolerance by antigen-encapsulated red blood cells.
| العنوان: | Innovative approach in Pompe disease therapy: Induction of immune tolerance by antigen-encapsulated red blood cells. |
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| المؤلفون: | Cremel M; ERYTECH Pharma, Lyon, France., Guerin N; ERYTECH Pharma, Lyon, France., Campello G; ERYTECH Pharma, Lyon, France., Barthe Q; ERYTECH Pharma, Lyon, France., Berlier W; ERYTECH Pharma, Lyon, France., Horand F; ERYTECH Pharma, Lyon, France., Godfrin Y; ERYTECH Pharma, Lyon, France. Electronic address: ygodfrin@erytech.com. |
| المصدر: | International journal of pharmaceutics [Int J Pharm] 2015 Aug 01; Vol. 491 (1-2), pp. 69-77. Date of Electronic Publication: 2015 Jun 06. |
| نوع المنشور: | Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: Elsevier/North-Holland Biomedical Press Country of Publication: Netherlands NLM ID: 7804127 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-3476 (Electronic) Linking ISSN: 03785173 NLM ISO Abbreviation: Int J Pharm Subsets: MEDLINE |
| أسماء مطبوعة: | Original Publication: Amsterdam, Elsevier/North-Holland Biomedical Press. |
| مواضيع طبية MeSH: | Antigens/*immunology , Erythrocytes/*immunology , Glycogen Storage Disease Type II/*immunology , Immune Tolerance/*immunology, Animals ; Immunity, Humoral/immunology ; Mice ; Mice, Inbred C57BL ; alpha-Glucosidases/immunology |
| مستخلص: | Pompe disease is a glycogen storage disease caused by acid α-glucosidase enzyme deficiency. Currently, the unique treatment is lifelong enzyme replacement therapy ERT with frequent intravenous administration of the recombinant analog alglucosidase-α (AGA), which ultimately generates a sustained humoral response resulting in treatment discontinuation. Our aim is to use the tolerogenic properties of antigen-encapsulated red blood cells (RBCs) to abolish the humoral response against AGA and to restore tolerance to replacement therapy. To demonstrate that our approach could prevent the AGA-induced immune response, mice were intravenously injected three times with AGA encapsulated into RBCs before being sensitized to AGA with several adjuvant molecules. Control animals received injections of free AGA instead of the encapsulated molecule. One-week after treatment with AGA-loaded RBCs, a strong decrease in specific humoral response was observed despite three stimulations with AGA and adjuvant molecules. Furthermore, this specific immunomodulation was maintained for at least two months without affecting the overall immune response. AGA-loaded RBCs represent a promising strategy to induce or restore tolerance in Pompe disease patients who develop hypersensitivity reactions following repeated AGA administrations. (Copyright © 2015 Elsevier B.V. All rights reserved.) |
| فهرسة مساهمة: | Keywords: Alglucosidase-α; Antibodies; Pompe disease; RBCs; Tolerance induction |
| المشرفين على المادة: | 0 (Antigens) EC 3.2.1.20 (GAA protein, human) EC 3.2.1.20 (alpha-Glucosidases) |
| تواريخ الأحداث: | Date Created: 20150610 Date Completed: 20160505 Latest Revision: 20150805 |
| رمز التحديث: | 20221213 |
| DOI: | 10.1016/j.ijpharm.2015.05.062 |
| PMID: | 26056928 |
| قاعدة البيانات: | MEDLINE |
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