Gene therapy was once lauded as the next revolution in medicine, but for many years clinical success was elusive and pessimism towards gene therapy emerged. However, two decades of progress in vector development, and insights gained from pre-clinical and clinical studies have now yielded breakthroughs in gene therapy for a number of diseases, including genetically inherited immune deficiencies, metachromatic leukodystrophy, the bleeding disorder hemophilia B, a form of hereditary blindness, and even cancer.
We are developing new strategies to induce antigen-specific immunological tolerance in order to prevent the autoimmune attack that causes type I diabetes. To do this we are employing a novel gene-based approach to target antigens to tolerogenic cell types by exploiting the microRNA regulatory network of these cells (Brown et al. Nat Med 2006; Annoni, Brown et al. Blood 2009). This targeting strategy will help us to identify cell types and cell states that can direct T cells to become regulatory T cells.