What is it about?
The delivery of genes or RNA interference (e.g. siRNA) into specific cells, for the expression or knockdown of specific gene products, is essential for the exciting prospects of gene therapy. However, optimal gene therapy delivery systems are lacking. We have developed a peptide-based, multicomponent platform for the delivery of siRNA/pDNA, which incorporates: 1) a poly-L-lysine dendron for DNA/RNA condensation, 2) a cell penetrating peptide for cell uptake, and 3) and endosome escape species to improve the delivery of siRNA/pDNA into the cytosol. In this publication, we extend this system to include a fatty acid, which is found to generate a system with the capacity to efficiently deliver siRNA to knockdown genes of interest, and which demonstrates a lack of toxicity.
Why is it important?
The addition of a fatty acid to this system creates an efficient platform for the deliver of siRNA. Such platforms will be essential for the future success of gene therapy.
The following have contributed to this page: Dr Peter Michael Moyle, Professor Istvan Toth, and Yu Wan