Treating inherited immune defects
The key to success in treating patients who develop immune deficiency is the ability of clinicians to work alongside scientists who have insights into particular parts of the immune system.
Bone marrow transplantation for PID
As bone marrow transplant (BMT) consultants and scientists at UCL, we have been working with our colleagues at the IIT to develop a bespoke clinical and scientific transplant programme for adult primary immunodeficiency (PID) patients.
As these diseases are so rare, the complex, novel transplants are performed only after detailed consideration.
We also carry out stem cell gene therapy to correct serious defects in immunity, previously used only in babies and children. We use detailed genomic analysis, studies of the mechanisms of the immune system and combine these with comprehensive clinical data to inform decisions about patient care.
By bringing together researchers and the clinical teams in one space, the Pears Building accelerates and supports these critical interactions.
Gene therapy for PID
I run a research team at the IIT focusing on understanding the underlying causes of PID.
Our team focuses on improving early diagnosis and treatment and works closely with the largest clinical service for people with PID in the UK. We have treated the largest group in the world of adult PID patients with allogeneic BMT and are the only team to have treated two older adults with gene therapy for PID.
In collaboration with Professor Emma Morris, I have expanded my research team. Together, we are developing new gene editing approaches to treat PID for patients not eligible for BMT.
The new Pears Building provides the space and equipment to attract world class scientists to join our team and help our patients to access potentially curable cutting edge therapies.
Transplant immunology
My lab focuses on understanding the key switches that regulate whether or not we make an immune response. If we make an immune response too easily, we risk triggering autoimmune diseases such as rheumatoid arthritis or type 1 diabetes.
My team works on a protein called CTLA-4 which acts like a tiny vacuum cleaner, removing proteins that stimulate the immune system. If CTLA-4 is faulty the immune system gets overactive.
At the IIT we work with doctors who treat patients with defects in the CTLA-4 gene. By applying basic scientific knowledge we’re able to help diagnose the problems. Studying patients has also helped us to learn more about how this key system works.
The Pears Building provides outstanding new laboratories for our work and improves the opportunities for us to learn about how the immune system is controlled.
