With 1 in 20 children born in Australia suffering from some kind of chronic disease or congenital abnormality like muscular dystrophy, epilepsy or cystic fibrosis, 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.

It is through understanding the most basic inner workings of our cells that researchers aim to find the insight needed to advance the prevention and treatment of many diseases that rob children of the healthy life that should be their birthright.

The Gene Therapy Research Unit at the Children’s Medical Research Institute was established in recognition of the exciting potential of gene therapy. Working in partnership with the Children’s Hospital at Westmead, the Unit conducts fundamental genetic research to understand the genes important for health and development, and the underlying causes of disease.

Researchers study the genes and molecules that make our bodies’ cells grow and develop from the moment of conception onwards, and the genes that maintain the healthy function of our bodies. By studying the key changes that occur during cell development researchers can identify when, where and how faults may occur. If small problems can be detected at this stage, they could be prevented from becoming big problems or treated to reduce their impact on children’s quality of life.

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.

For more information visit:  http://www.cmri.org.au/