Institute of Biomedical and Biomolecular Science (IBBS)

Molecular and genetic medicine

Gene therapy

The second area of our research interest includes development of non-viral gene therapy vectors and methods for targeting of therapeutic DNA into skeletal muscle. In collaboration with chemists we are developing and studying the properties of novel polymeric vectors (Lavigne et al, 2005, 2006; Pennadam et al, 2004a,b, 2007; Twaites et al, 2004, 2005).

Pluronic block co-polymers find application in various areas of medicine and are promising agents for  gene targeting using non viral vectors. However, this enhancement of transgene expression with pluronic is promoter-specific: Only CMV–driven constructs are affected. Therefore pluronics may act as biological response modifiers, influencing expression of endogenous genes. Effects of such gene activation may, potentially be both beneficial and harmful - therefore we are involved in studies aiming to understand this effect.

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Fig. 1 - Pluronic SP1017 only enhanced expression of CMV promoter-driven plasmid vector (black bars) following targeting into muscle (For more details see: Lavigne et al, 2005 and 2008).