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Gene therapy to discover new type 2 treatments

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. 

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.  

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. 

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. 

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.  

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.  

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.  

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.  

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.  

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