Gene Therapy – The Answer to Childhood Genetic Diseases?

The need for effective treatments for children with rare genetic diseases has been a long and challenging road in research and the community. With the advancement of technology, research is now a step closer to an answer – gene therapy.

A major game changer in medicine, gene therapy was recently addressed at the National Press Club of Australia by Dr Elizabeth Finkel, who is the first journalist to receive the Australian Society for Medical Research medal. Dr Finkel is the author of two books about stem cell and genome revolutions and co-founder of the popular science magazine Cosmos. For the full article published in The Conversation, click here.

What is Gene Therapy?

Gene therapy is a process that introduces genetic material into cells to replace or compensate for abnormal genes and/or to make a beneficial protein.

More specifically, modified viruses such as adeno-associated virus (AAV) and lenti virus are used as vectors to transport the gene replacement DNA to the target tissue and then the protein production.

For example, if a patient’s genetic disease is caused by too little of a particular protein, gene therapy may be used to increase the amount of that protein in the body.

Gene Therapy Research Unit

A joint initiative of Kids Research, Sydney Children’s Hospitals Network (SCHN)and Children’s Medical Research Institute, , the gene therapy research unit aims to develop innovative treatments for genetic diseases with focus on the therapeutic potential of rapid advances in biomedical knowledge.

The gene therapy research unit led by Professor Ian Alexander, a senior staff specialist and clinical geneticist at the Children’s Hospital at Westmead was the first group in Australia to treat a genetic disease, (SCID-X1, a rare immune deficiency disorder) by gene therapy.

Professor Alexander works collaboratively with Dr Leszek Lisowksi, a vectorology expert and group leader of his independent research program, the Translational Vectorology Group (TVG). They are engineering the next generation of vectors with objectives to improve delivery to the target organs and produce therapeutic levels of proteins.  

“Therapeutic potential of progress in the laboratory is our ‘raison d’etre’, so we are particularly pleased to have three clinical trials under development or close to initiation. This is a major achievement and places our group at the forefront of this exciting field in Australia.” – Professor Ian Alexander.

Contact us for advice and assistance on paediatric gene therapy clinical trials