Abstract
The present invention relates to nucleic acid expression cassettes and vectors containing liver- specific regulatory elements and codon-optimized factor IX or factor VIII transgenes, methods employing these expression cassettes and vectors and uses thereof. The present invention is particularly useful for applications using liver-directed gene therapy, in particular for the treatment of hemophilia A and B.
| Original language | English |
|---|---|
| Patent number | WO2016146757 |
| Publication status | Published - 2016 |
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Dive into the research topics of 'Optimized liver-specific expression systems for fviii and fix'. Together they form a unique fingerprint.Projects
- 1 Finished
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IOF1642: Financing IOF-program GEAR: Hyperactieve Expressie-modules
VandenDriessche, T. (Administrative Promotor) & Chuah, M. (Co-Promotor)
1/04/14 → 31/03/20
Project: Fundamental
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