This is one of several projects available on an MRC funded 4-year multi-disciplinary PhD programme in Human Genetics, Genomics and Disease at the MRC Human Genetics Unit (HGU), part of the Institute of Genetics and Molecular Medicine (IGMM) at the University of Edinburgh.
The recent game-changer for genetic disease is the utilization of CRISPR technology for human genome editing, thus offering a possible cure for genetic diseases. There are, however, sizeable obstacles to making this a therapeutic reality, including (a) targeted delivery of editing machinery and (b) improving repair efficiency.
To fast-track solutions to these problems, we developed powerful fluorescent reporter mouse models, allowing sensitive read-outs of genome-editing. In this studentship, you will determine whether differences in DNA repair pathways exist in different cell types and/or at different stages of differentiation, and whether these change with different delivery systems. You will help determine the most effective formulation for establishing therapeutically-beneficial editing in vivo.