Monash University > News and Events > Monash Memo

Gene therapy treats autoimmune disease

4 March 2009

Monash researchers have used a gene therapy strategy to treat autoimmune disease.

Autoimmune diseases such as multiple sclerosis and type 1 diabetes affect approximately five per cent of the population as a result of an individual's immune system attacking its own tissues.

Scientist from the University's Centre of Inflammatory Diseases Dr James Chan and Prof Ban-Hock Toh and Associate Professor Frank Alderuccio from the Department of Immunology have developed a strategy that involves the isolation and genetic manipulation of bone marrow stem cells together with bone marrow transplantation to promote tolerance by the immune system.

"Bone marrow stem cells are routinely isolated from humans for various disease treatments, and in this approach they would be used to promote tolerance,” Associate Professor Alderuccio said.

"We believe it offers a viable strategy of manipulating the immune system to remove or control autoreactive cells that would normally be involved in inducing disease.

Trials highlighted a resistance to disease following the transfer of genetically modified bone marrow stem.

"We were even able to incorporate this concept into a strategy to reverse established disease,” Associate Professor Alderuccio said.

"While still early days, this finding is very exciting and important when considering potential clinical application for human autoimmunity.”

This research has been published in the prestigious American Society of Immunology publication, Journal of Immunology.