Fakultäten » Medizinische Fakultät » Kinderspital Zürich: Medizinische Klinik » Immunologie, Abteilung » Prof. Dr. Reinhard Seger (emeritiert) » Siler
| Title / Titel | Monitoring of pediatric CGD gene therapy patients | ||
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| Abstract (PDF, 14 KB) | |||
| Summary / Zusammenfassung | Presently our laboratory is able to analyze blood and bone marrow samples of adult and infant CGD gene therapy patients by following assays: • FACS analysis of gp91phox • Analysis of gp91phox – p22phox assembly within the NADPH oxidase (Cytophrome B reduced minus oxidized difference spectrum) • Qualitative detection of respiratory burst activity (NBT reduction, DHR 123 oxidation, Chemiluminescence assay) • Quantitative analysis of respiratory burst activity (Cytochrome C reduction assay) • E.coli killing assay • A.fumigatus killing assay • Quantification of the percentage of gene modified cells in bone marrow stem/progenitor cells (colony assay combined with PCR based transgene detection in individual colonies of the colony assay). • NET formation assay Since 2005 we were able to monitor adult an infant patients after clinical CGD GT. |
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| Publications / Publikationen | Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006; 12: 401-9. M.G.O., M.S., K.S., S.S. and U.S. contributed equally to this work C.v.K., R.S. and M.G. share senior authorship |
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| Keywords / Suchbegriffe | Gene Therapy, CGD, NADPH oxidase | ||
| Project leadership and contacts / Projektleitung und Kontakte |
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| Funding source(s) / Unterstützt durch |
No project-specific funding |
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| In collaboration with / In Zusammenarbeit mit |
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| Duration of Project / Projektdauer | Apr 2005 to Oct 2011 |