Hossle
Fakultäten » Medizinische Fakultät » Kinderspital Zürich: Medizinische Klinik » Immunologie, Abteilung » Prof. Dr. Reinhard Seger » Hossle
Completed research project
| Title / Titel | Natural tissue-specific gene regulation after therapeutic gene transfer in chronic granulomatous disease (CGD) | ||||
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| Abstract (PDF, 14 KB) | |||||
| Summary / Zusammenfassung | This proposal is complementary to our ongoing projects, which are directed at the implementation of clinically applicable protocols for somatic gene therapy of X-linked chronic granulomatous disease (X-CGD) in children. Current Viral vectors have become very efficient gene delivery and expression systems for gene therapy. However the task of most natural truly tissue-specific regulation of transgene expression has not been achieved yet, but remains highly desirable. The delivered transgenes not only should be stably expressed at high levels when appropriate, but at the same time, misplaced expression in time or location needs to be avoided. Unfortunately, like for other natural endogenous promoters, the endogenous regulatory elements required for tissue-specific expression of our transgene of interest, the gp9Iphox gene, are too large to be included in a vector construct for gene therapy purposes. Therefore, combinations of alternative regulatory elements should be constructed and evaluated for their power to drive transgene expression most similarly to physiologic myeloid-specific gp91phox expression. | ||||
| Project leadership and contacts / Projektleitung und Kontakte |
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| Funding source(s) / Unterstützt durch |
Foundation CGD Research Trust, UK |
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| Duration of Project / Projektdauer | Jul 2000 to Dec 2002 |