A gene therapy for diabetes can be effective by targeting the liver, Italian researchers reported last night.
The scientists chose the liver because it is thought to be able to suppress the immune system.
Their aim was to prevent the immune system reactions which lead to the destruction of insulin-producing cells in the pancreas.
Reporting in Science Translational Medicine, the researchers say how they successfully triggered the liver to mobilise regulatory T cells. These destroyed the rogue immune cells that damage the pancreas.
The therapy has so far been tested in laboratory mice and has restored blood glucose in these animals to normal levels, the researchers say.
It uses a harmless virus to deliver a gene with a fragment of the insulin antigen, the insulin B chain 9-23.
Researcher Mahzad Akbarpour, from San Raffaele Scientific Institute in Milan, Italy, writes: "With further development, this new antigen-based gene therapy can potentially help restore immune tolerance at the onset and advanced stages of type 1 diabetes."
Insulin B chain 9–23 gene transfer to hepatocytes protects from type 1 diabetes by inducing Ag-specific FoxP3+ Tregs. Science Translational Medicine 27 May 2015 [abstract]
Leave a Reply