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Gene therapy to target T cells in Type 1

Project summary

Targeting islet-specific CD8 T cells in Type 1 diabetes

In Type 1 diabetes, T cells of the immune system destroy insulin-producing cells in the pancreas. Professor Susan Wong will work to develop a gene therapy to encourage the body’s own immune system to destroy the ‘bad’ T cells and protect against Type 1 diabetes.

Background to research

White blood cells of the immune system usually help to protect against infection by targeting bacteria and viruses that invade the body. But, in Type 1 diabetes a small number of these cells, called ‘killer’ T cells, target and destroy the insulin producing cells in the pancreas by mistake.  Gene therapy, which introduces genes into cells in order to deliver targeted therapy, is already undergoing clinical trials for the treatment of cancer. With support from Diabetes UK, Professor Susan Wong and her team have shown that cells from a genetically-manipulated mouse can be used to target the specific killer T cells that cause diabetes, and protect mice from the condition. 

Research aims

Professor Wong will build on her previous work funded by Diabetes UK, to move closer to a gene therapy for people with Type 1 diabetes. The aim of the therapy is to introduce genes into cells of the immune system and reprogram them to target the specific ‘killer’ T cells that cause Type 1 diabetes. This technique should selectively target and destroy ‘bad’ immune cells that cause Type 1, without harming the ‘good’ immune cells, which help to protect against infection. The genes are introduced temporarily and do not affect the main genetic code of cells, which should make the therapy safer and allow this work to move towards human trials more quickly. The researchers aim to find out if this therapy is effective at selectively targeting and removing the ‘bad’ T killer cells and if it can prevent Type 1 diabetes in mice.

Potential benefit to people with diabetes

This work is at a relatively early stage but, if successful, could potentially be tested in humans in the next five to ten years. If the therapy works well in the early stages of Type 1, it could provide a potential strategy for preventing the condition. And if it works well in the later stages after diagnosis, then it might help to protect surviving islet cells or transplanted islets from further immune attack.

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