Investing in the health of future generations of Australian children

With 1 in 20 children born in Australia suffering from some kind of chronic disease or congenital abnormality like muscular dystrophy, epilepsy or haemophilia, gene therapy is providing renewed hope of treatments being developed for these inherited childhood diseases. With the potential to correct the genetic basis of disease, gene therapy - the use of genes as medicine, represents one of the most exciting prospects in medical research.

The Gene Therapy Research Unit (GTRU), a collaboration between the Children’s Medical Research Institute and the Children’s Hospital at Westmead, was the first of its kind in Australia.

 
Gene Therapy for inherited childhood disorders aims to correct the cause of a condition by delivering a healthy copy of a gene to the patient’s cell in order to compensate for a missing or damaged gene.

The GTRU at the Children’s Medical Research Institute recently sought the support of the Honda Foundation to purchase essential specialist equipment for their research laboratories. They needed a PCR Machine to support and enhance their current ground breaking research. Polymerase chain reaction (PCR) is a technique that allows researchers to amplify the quantity of DNA in a sample to enable detailed analysis of genes and gene mutations that cause disease.

The GTRU is conducting research into gene therapy for childhood liver diseases. The team is also working on a therapy for the immune disorder "Severe Combined Immunodeficiency". This condition is also known as "Boy in a Bubble" disease due to the sterile isolated environment these little boys must suffer to protect them from common ailments such as colds and flu that could kill them. A third project in the laboratory is into gene delivery to treat infectious disease and immune disorders.

The Honda Foundation with the support of Trivett Honda was delighted to approve funding of $10, 230.00 for the purchase of the PCR Machine for the GTRU, to assist them to identify new avenues of treatments for a range of childhood diseases.

Dr Ian Alexander, Head of the GTRU said "This PCR machine is fundamental to the majority of experiments that we do in the laboratory. So the support of the Honda foundation is very much appreciated. It will greatly enhance the progress of our research. Thank you for helping us work towards a healthier life for all children."