Genomic Engineering Group

For over 100 years, experimental mice have been the major in vivo system used to investigate the molecular, cellular and genetic basis of health and disease. Over the past 30 years, the ability to make specific alterations to the mouse genome and produce ‘genetically modified’ mice has significantly advanced research practices. Two new technologies, TALEN and CRISPR, are revolutionising this process by making single point mutations or multiple gene modifications in mice possible in a rapid, cost-effective manner.

We are using advancements in creating genetically modified mice, along with our increased understanding of the genetic basis of disease, to create novel mouse models of human disease. These animal models will further our understanding of the aetiology of disease and also help develop therapeutics for the treatment of disease.

The development of precise genome editing technology, such as CRISPR – base or prime editing –has the potential to cure human disease by the genomic correction of disease-causing mutations. Our lab is developing the technology to extract haematopoietic stem cells in genetically modified diseased mice, correct disease mutations with CRISPR and then return those cells to the mice to restore health. Our ultimate objective is to develop systems that will deliver, in vivo, highly specific gene modifying machinery to specific cell types, to correct disease-causing mutations.