England (Commonwealth Union) – Gene editing has played a key role in medical and agricultural biotechnology, to fight diseases and genetic engineering of crops. Researchers at University College London (UCL) have found a fault in cells that form a significant component of the immune system that can be fixed with ground breaking gene editing methods, seen in a new study in human cells and mice.
Researchers say the study, can pave the way for new treatments for a rare disease of the white blood cells that generally regulates the immune system (regulatory T cells), and those that defend against repeat infections and cancer (effector T cells). Patients lacking CTLA-4, carry mutations in a gene leading T cell to function abnormally. It brings about severe autoimmunity, where their immune system causes self-destruction attacking its own organs, tissues and blood cells.
The human cell, use of ‘cut’ and ‘paste’ gene editing methods, with CRISPR/Cas system, where the scientists were capable of targeting the faulty gene in T cells obtained from patients with CTLA-4 insufficiency and fix the errors. This stabilized the amounts of CTLA-4 in the cells to those seen in healthy T cells. The scientists were also able to enhance symptoms of the disease in mice with CTLA-4 insufficiency by giving them injections of fixed gene edited T cells.
Co-senior author, Professor Claire Booth, Mahboubian Professor of gene therapy and paediatric immunology at UCL Great Ormond Street Institute of Child Health, said: “Our approach has many positive aspects. By correcting the patient’s T cells, we think it can improve many of the symptoms of the disease, at the same time as being much less toxic than a bone marrow transplant. Collecting the T cells is easier and, correcting the T cells is easier.” She further stated that with this approach the hospital time for patients will be lowered.
