New Gene Editing Strategy Could Lead to Treatments for People Born with Inherited Diseases of Immune System

A fault in cells that form a key part of the immune system can be repaired with a pioneering gene editing technique, finds new research demonstrated in human cells and mice, led by University College-London (UCL) scientists. Researchers say the study, published on October 26, 2022 in Science Translational Medicine, could lead to new treatments for a rare disease of the white blood cells that normally help to control the immune system--known as regulatory T cells--and those that protect the body from repeat infections and cancer--known as effector T cells. The article is titled “Therapeutic Gene Editing of T Cells to Correct CTLA-4 Insufficiency.” Patients with the condition, known as CTLA-4 insufficiency, carry mutations in a gene that cause these T cells to function abnormally. It leads them to suffer from severe autoimmunity, where their immune system attacks their own tissues and organs, including their blood cells.