Nonviral Vector Development
Plasmid DNA molecules are one of the most widely used tools in molecular biology research. They are derived from naturally occurring molecules in microbes.
These circular double stranded DNA molecules have been manipulated by molecular biologists to allow for the propogation and study of interesting pieces of DNA from a wide variety of sources. Plasmid molecules are available to meet a wide range of needs.
Whether the plasmid is required for a small piece of work, or if it is intended for future clinical use, the process always starts by transforming the molecule into E. coli so it can be replicated by the microbial machinery.
Restriction endonuclease enzymes are used cut up DNA and individual fragments can be readily visualised and purified on gels. This has allowed us to create a series of modular plasmid molecules comprised of interchangeable bacterial bacbones, promoter/enhancer regions and transgenes.
This has allowed our group to optimise the elements within our plasmids so they are suitable for clinical use.
We have developed a range of promoter elements that direct long-term expression in-vivo and have reduced the toxicity of the plasmid by removing elements which cause inflammation in human cells.
This process culminated in the development of our current clinical trial plasmid, pGM169.
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Medical Futures Innovation Award 2011