Functional genomics and development of clinical genome editing strategies

The Davies laboratory aims to understand how the genome is read by the cells of the blood and bone marrow and how the sequence can be edited to treat human disease. This is important because genetic variation in bloodcells alters the susceptibility to many different human diseases including infection, autoimmune disease and malignancy. Genome editing of these cells has the potential to cure many diseases but this technology can only be safely used if we understand the effects of altering the genome sequence. Over 90% of all DNA sequences that are associated with human disease alter how genes are controlled rather than in the protein produced by the gene. The laboratory has world class expertise in developing new techniques for determining the physical structure of DNA within cells, which is key to understanding how the genome functions. We will use this technology to investigate the fundamental principles that control genes. We will also use it to map the key DNA sequences that control genes in important blood and bone marrow cell types. We aim to translate this by developing new and safer ways of editing the genome of bone marrow stem cells which can be transplanted to treat human disease.