Gene Therapy and Gene Editing for Very Early Onset Inflammatory Bowel Disease.

Individuals with mutations in the receptor for IL-10 suffer from very early onset Inflammatory Bowel Disease. These patients are typically unresponsive to conventional therapies and have a poor long-term prognosis. The only available curative therapy is bone marrow transplantation from a suitable donor, which carries significant risks and often is not available. We propose to use gene transfer into the patient’s own stem cells to restore IL-10 signaling and provide an alternative treatment option for these patients. Our preliminary studies indicate that this gene therapy approach can prevent the development of the disease in a mouse model which mimics the disease symptoms observed in human patients. The team of investigators has deep expertise covering the diverse aspects of this project, with expertise in gene therapy preclinical development, clinical translation and in Inflammatory Bowel Disease and IL-10 biology.