OUR TECHNOLOGY

We have developed gene regulatory compounds, known as DNA-binding polyamides (DBPAs), that recognize SNPmt DNA sequences in the COXIII gene which is associated with a range of neurodegenerative disorders. These candidates can alter the expression the malfunctioning protein by blocking transcription of the diseased gene, whilst potentially not compromising the expression of ‘healthy’ wildtype (WT) sequences. We have also shown that these gene regulatory compounds can be delivered to into cells and into the mitochondria where the genetic target is located. Our gene regulatory compounds have modified components that enhance selectivity for SNPs over WT sequences, thus reducing the propensity for off-target adverse effects.