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Improvement of rAAV5 transduction efficiency in fibroblast-like synoviocytes by doxorubicin
  1. C J Aalbers,
  2. S A Loiler,
  3. I de Vries,
  4. F Mingozzi,
  5. M J Vervoordeldonk,
  6. P P Tak
  1. AMC/University of Amsterdam, The Netherlands

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Background

We are currently developing local gene therapy for the treatment of rheumatoid arthritis (RA) using interferon β (IFNβ) as therapeutic gene and rAAV5 as vector. We generated a rAAV5 vector that expresses hIFNβ under control of an inflammation inducible promoter (rAAV5.NFkB.hIFNβ). To support development of our clinical gene therapy programme a robust assay is needed to transduce different cell lines with rAAV5.NFkB.hIFNβ. In vitro, rAAV5 does not transduce cells efficiently, including fibroblast-like synoviocytes (FLS), the main target cell …

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