Oligonucleotides as therapeutic agents

Designing new cures for diseases is the goal of most chemical biologists and with this in mind we are designing and synthesising modified oligonucleotides as potential therapeutic agents. In this context we are developing methods of reversing the mutations that give rise to genetic diseases. The basis of this research is to initiate DNA repair by the formation of triple helices at the site of the mutation. Triple helix forming oligonucleotides (TFO's) have wider applications in molecular genetics, diagnostics, therapeutics and nanotechnology, and in collaboration with Prof. Keith Fox in Biological Sciences in Southampton we have developed a set of four base analogues that, when incorporated into TFOs, are able to recognize mixed sequence DNA with high specificity. This is the first example of full sequence recognition of a DNA in the major groove (View paper).