Marianne Rots is professor in Molecular Epigenetics and her team aims to target epigenetic mutations to restore gene expression profiles.
Epigenetics refers to the study of heritable changes in gene expression that occur without a change in the sequence of the DNA. DNA methylation and posttranslational modifications of the histones (DNA-packaging proteins), are well known for affecting gene expression levels. For an increasing list of diseases, abnormalities in gene expression have been linked to aberrant levels of DNA methylation and disturbed profiles of histone modifications (epimutations).
In contrast to genetic mutations, epimutations are reversible. Current drugs targeting epigenetic enzymes indeed show re-expression of silenced genes, but such drugs act non-selectively and transiently. The innovative technology of Epigenetic Editing circumvents such limitations by engineering sequence-specific DNA binding domains to achieve gene-specific targeting in the human genome. These agents then are fused to epigenetic enzymes in order to permanently overwrite the epigenetic signature specifically at the site of interest (see also our recent review).
This research, financed among others by NWO (VIDI, ChemThem), EU FP7 (SNN) and the UMCG (RFF), fully exploits the druggable genome concept and will open up new avenues for biomedical research and therapeutic applications.
|Last modified:||14 September 2016 3.10 p.m.|