Regulation of gene expression for improved therapies
Fundación para la Investigación Médica Aplicada (FIMA/CIMA), Navarra, Spain
Genes can be delivered to patients for the treatment of hereditary disorders and a myriad of multigenic diseases including cancer. This is the basis for gene therapy, expected to transform the health system in the near future. Before these therapies can be widely implemented, it is compulsory to have a technology that sustains the level of the therapeutic gene within the threshold.
The team has created ROUTER, an inducible system based on a RNA sequence that, when accessible, interacts with a cellular complex that blocks gene expression. The system reaches inducibility when a given drug binds a riboswitch structure that responds by hiding the complex binding sequence, leading to gene expression.
ROUTER 1.0 is small in size, invisible for the immune system, safe, cell-type independent, displays synergistic effects when multimerized and potent. ROUTER 2.0 will be developed to respond to FDA-approved drugs with no toxicity and excellent bioavailability.