Gene editing medicines can be utilized effectively to introduce sequence modifications, insertions, or deletions in the genome, offering a therapeutic strategy to treat human diseases by modifying protein sequences or protein expression levels. Our overall objective is to develop novel gene editing medicines for severe rare diseases and severe patient segments in common diseases with high unmet medical need. Identifying potential disease indications and targets requires multidimensional data including clinical measurements and health outcomes, as well as deep genomics and other molecular data. In this respect, the UK Biobank data will enable us to characterize the full range of clinical and molecular consequences of naturally occurring sequence variation and regulatory sequence disruptions in a large population cohort.
The potential impact of this research on public health is significant, as a primary application of our research will be to develop medicines for severe rare diseases, which collectively impact up to ~6% of the global population, with a high unmet medical need. A secondary impact is that as more gene editing medicines are developed, potential safety concerns will be better identified and the technical limitations will be addressed, making possible the expansion of gene editing medicines into more common diseases.