The new MRC CoRE in Therapeutic Genomics aims to transform the diagnosis and treatment of genetic disorders by enabling the mass development of cutting-edge genetic therapies.
Instead of creating standalone treatments for each disorder, we are building customisable “therapeutic platforms.” These are methods that can be adapted to treat many different disorders. We are creating a set of tools to change the genetic message, a set of vehicles to deliver the therapies, and knowledge to adapt and retarget them for new conditions. This toolkit will include data, computer models, expertise, delivery systems, assays and high-throughput processes for exploring and testing new therapies.
We will begin with a few example diseases that affect the blood, the eye and the brain. The lessons we learn from these will help us make treatments for other genetic disorders.
