Disease Causing Genes

Assassin technology: Shooting disease-causing genes

We are all living longer and the percentage of older people in our communities is growing. One of the effects of ageing on our population is a dramatic rise in chronic disease. Combating these age-related illnesses is at the core of Professor Levon Khachigian’s work. Khachigian, an NHMRC Australia Fellow, from the Centre for Vascular Research at the University of New South Wales, believes heart disease, arthritis, cancer and blindness all have something in common, and he has this link squarely lined up in his research riflescope.

Khachigian, a recent President of the Australian Society for Medical Research, believes an increasingly elderly society is one of our major health challenges. “The world’s population over the age of 65 will more than double by the year 2050,” he states. “Australia, and indeed, all APEC economies, face a rapid rise in the burden of chronic disease such as ischemic heart disease, diabetes, rheumatoid arthritis, and malignancies. This represents a serious and economic challenge to Australia and the international community, which our children stand to inherit.” Khachigian cites one sobering example. “There’s been a 90% increase in the global incidence of diabetes (45% in Australia) in the 15 years to 2010,” he says. “If you’ve had diabetes for 20 years, there’s a very strong likelihood you’ll have some form of retinopathy. Retinopathy and a related form of blindness – age-related macular degeneration – afflict millions worldwide,” he explains.

Khachigian, a vascular cell and molecular biologist, is finding new ways to treat these diseases by giving proteins that are known as ‘transcription factors’ a knockout blow. These proteins live in the nucleus of a cell and integrate genes with disease. “In simple terms, we’ve been targeting messenger RNA that encode harmful ‘master regulatory’ genes,” he says. “In this setting, it is good to shoot the messenger with our molecular assassin technology, because the mRNA we’re targeting form proteins, that if allowed to run wild, activate hoards of disease-causing genes.”

This research, funded by the NHMRC, hopes to uncover new insights into the roles of these proteins so that this new knowledge can be harnessed in the generation of novel therapeutic agents and fuel a vibrant drug development pipeline for many diseases.

Despite the challenges of our ageing world, Khachigian is optimistic about the outlook. “I’m just thrilled by what the future holds,” he says. He’s also passionate about the role of research in changing health outcomes. “Medical research is a ‘golden egg.’ It prevents disease, improves quality of life, reduces hospital stays and helps drive workforce productivity and wealth creation. The Australia Fellowship will fast track important discovery science and not only that, the Fellowship will build national capacity, and open up an array of exciting opportunities for the next generation of researchers.

“The NHMRC has provided me with critical oxygen that’s fuelled this passion, with vital program and project grant support and progression through a research fellowship scheme that both drives and is driven by excellence and is
the envy of the world.”

This article was originally published in Australian Health and Medical Research – Working to Build a Healthy Australia.