Project summary
There are a number of inherited genetic conditions causing very low body fat stores. One of these is congenital generalised lipodystrophy type 2 which can also lead to type 2 diabetes. Dr Mcilroy’s shown that gene therapy can treat this condition so he’s now going to study it in more detail. He’s hoping this knowledge will lead to better treatments for type 2 diabetes.Â
Background to research
People living with a very rare genetic condition called congenital generalised lipodystrophy are born with most of their body fat stores missing. People with the condition also develop type 2 diabetes. This shows the important link between body fat and the control of blood sugar levels. Â
Scientists have studied this link and found that giving fat back to mice with congenital generalised lipodystrophy could prevent them from developing type 2 diabetes. Dr Mcilroy has researched another successful way to treat this, using gene therapy. Gene therapy involves using a harmless virus to deliver a missing gene. He found that just a single treatment was needed to stop type 2 diabetes in the mice.Â
Research aims
Dr Mcilroy now wants to study this gene therapy further to see if it’ll provide clues for new treatments for type 2 diabetes.Â
He’ll start by finding out more about how the gene therapy works in mice with congenital generalised lipodystrophy and how it brings about benefits. Â
He’ll then aim to discover how the gene therapy affects blood sugar levels by looking at changes that the therapy brings about in the liver, which is a key organ involved in blood sugar control. He’ll do this by studying the gene activity in single liver cells, using cutting-edge technology. Â
This will find potential new pathways that could be targeted to treat type 2 diabetes. Dr Mcilroy will aim to find out which of these targets are most promising to prevent high blood sugar levels by studying them in mice who are at risk of type 2 diabetes. Â
Potential benefit to people with diabetes
This research could help to develop a pioneering new gene therapy that aims to prevent or reverse type 2 diabetes in people with a rare health condition. Â
Beyond this, these discoveries could pave the way for new treatments for people living with type 2 diabetes that tackle blood sugar levels in new, more effective ways. Â
