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31 Aug 2021

Prof Shane Grey to lead gene therapy for type 1 diabetes

Garvan immunologist Professor Shane Grey has received critical funding to lead research into a new treatment approach for type 1 diabetes.

To continue his pioneering work in finding new approaches to treat type 1 diabetes (T1D), Professor Shane Grey has been awarded a significant grant by JDRF, the leading global funder of type 1 diabetes research.

He will lead a collaborative team to develop viral vectors aimed at protecting insulin-producing cells in the pancreas from immune destruction and thereby restoring production of insulin – a hormone that is central to controlling blood sugar regulation. If successful, the approach could reduce T1D patients’ reliance on daily injections and help prevent some of the significant disease side effects.

Professor Grey, who heads the Transplantation Immunology Lab at the Garvan Institute of Medical Research, will be collaborating with Associate Professor Leszek Lisowski from the Children’s Medical Research Institute’s Translational Vectorology Unit to carry out the research.

“This JDRF funding is critical for allowing us to pioneer a new therapy approach for T1D, 2800 cases of which are diagnosed each year in Australia alone. This project will continue our work on pancreatic beta cells and whether boosting a protective molecule called A20 within these cells could partially restore their insulin-secreting function,” says Professor Grey.

“We look forward to applying our expertise in viral vector-based gene therapies to explore new therapeutic options for patients with T1D. The extensive combined basic and translational research experience of our research teams is an exciting prospect for the improvement of those affected by the disease,” says Associate Professor Lisowski.

New approach for a chronic condition

T1D is a chronic autoimmune disease where the body attacks the beta cells in the pancreas that make insulin. Insulin is the hormone that manages blood glucose – a major fuel source for our bodies. A deficiency in insulin can lead to hyperglycaemia, which can cause organ damage.

While individuals affected by T1D must regularly monitor their sugar levels and inject insulin several times a day, even those who carefully manage the condition can suffer from serious complications that can reduce life expectancy.

“In our project, we aim to restore the ability of beta cells to produce insulin,” explains Professor Grey. “In our previous research, we discovered that the immune molecule A20 confers protection to beta cells and can prevent their degradation. In this project, we will determine if boosting A20 within beta cell can restore their function.”

A central component of this project is the collaboration with Associate Professor Lisowski from the Children’s Medical Research Institute, who has expertise in bioengineering adeno-associated viral (AAV) vectors, a promising form of gene therapy that aims to target cells in patients and deliver genetic material that modifies their function.

The researchers aim to develop the viral vectors to target human beta cells and deliver the A20 gene for production within these cells. The team will then validate their vectors in preclinical studies using experimental models, a crucial first step in translating the potential therapy to patients.

Professor Grey is a Conjoint Associate Professor at St Vincent's Clinical School, Faculty of Medicine and Health, UNSW Sydney.