Gene Therapy

Researchers from University of London, Imperial College detail findings in gene therapy

Published in Blood Weekly, February 5th, 2009

"An ideal gene therapy vector should enable persistent transgene expression without limitations of safety and reproducibility. Here we report the development of a non-viral episomal plasmid DNA (pDNA) vector that appears to fulfil these criteria," investigators in London, the United Kingdom report.

"This pDNA vector combines a scaffold/matrix attachment region (S/MAR) with a human liver-specific promoter (alpha 1-antitrypsin (AAT)) in such a way that long-term expression is enabled in murine liver following hydrodynamic injection. Long-term expression is demonstrated by monitoring the longitudinal luciferase expression profile for up to 6 months by means of in situ...

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Source: Blood Weekly (2009-02-05)

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