Our translational research programs are bringing together cutting-edge genetic tools and insights from basic immunological research, to create transformative cures for pediatric diseases.
Instead of kids needing lifetime treatment, researchers at the Center for Immunity and Immunotherapies (CIIT) are working to create new cell therapies, removing a small number of cells — either stem cells or immune cells like T or B cells — from a patient’s blood or bone marrow, and then changing the genetic instructions in those cells to reprogram them. The scientists are also developing new ways to perform gene therapy directly within the body to make the process more efficient and less expensive, generating new methods for delivery into the bloodstream or the bone marrow to create cell therapies immediately within the body for treating diseases ranging from hemophilia to HIV.
CIIT researchers also use gene editing to create regulatory T cells that calm the immune system in children with diabetes and other autoimmune diseases and in children who need organ transplants.
Our immunology experts are learning how to harness regulatory T cells to act like peacekeepers in these patients so the overprotective immune system doesn’t cause harm.
Antibody-secreting B cells live for decades in our bodies and can produce thousands of protein molecules per second to protect us from infection. CIIT’s immunology scientists are taking advantage of these unique cell qualities and using gene editing to create B cells that can produce large quantities of therapeutic proteins. B cell therapies can transform the delivery of a missing protein. The cells become a “living therapy” given to patients in one shot to stop diseases, like primary immune deficiency, rheumatoid arthritis, and lupus long term.