Using gene technology in human health
Gene technology is providing breakthroughs in human health that were previously impossible. Some of the uses of genetically modified organisms (GMOs) in human health include:
- producing vaccines or medicines
- developing diagnostic tests
- treating genetic diseases using gene therapy
- developing targeted therapies for diseases such as cancer.
Genetically modified medicines
There are currently 2 types of medicines that use gene technology. These are:
- medicines made from GMOs – for example, using genetically modified (GM) bacteria to produce insulin
- GMOs used as medicines – for example, Gardasil vaccine to prevent human papillomavirus, and the mRNA COVID-19 vaccines (Comirnaty (Pfizer) and Spikevax (Moderna)).
Scientists are also researching the development of GM plants that can produce edible vaccines – where the body absorbs the vaccine when the food is eaten. The advantages of these include not needing:
- an injection, making them easy to administer
- special storage facilities or sterile needles.
Using gene technology in medical therapies
An example of GMOs as medicines is gene therapy. Research shows that gene therapy can potentially treat genetic diseases, cancer and infectious diseases, and can produce vaccines.
At its simplest, gene therapy involves inserting DNA or RNA into cells to treat or prevent diseases that are not treatable with medicines.
This is only done to somatic cells (that cannot be inherited) so any changes made to a person’s cells will not be passed on to future generations. Making changes to human genes that are inheritable is currently banned under the Prohibition of Human Cloning for Reproduction Act 2002 in Australia.
This is done by either:
- replacing a faulty gene with a healthy one
- ‘turning off’ a faulty gene that is causing a disease or condition
- adding a new gene to treat a disease or symptoms of a condition – either a gene that is missing or one that introduces new functionality.
For example, scientists have:
- introduced new genes into leukaemia sufferers that make it easier to target and destroy the cancerous cells
- developed a single dose gene replacement treatment for spinal muscular atrophy.
The Therapeutic Goods Administration (TGA) have only approved a limited number of gene therapy treatments for use in Australia. In urgent medical situations, doctors can apply to the TGA’s Special Access Scheme. Applications are for a single patient, and the TGA assesses these on a case-by-case basis.
How the scheme affects human health
The TGA regulates all therapeutic goods available in Australia, such as medicines, medical devices or biologicals. This includes all dealings with live, viable GMOs, such as those intended:
- for use as medicines
- to manufacture medicines
- to test medicines.
When the TGA receives an application for therapeutic goods that contain GMOs, it must consult the Office of the Gene Technology Regulator (OGTR). The OGTR administers the Gene Technology Act 2000 and keeps records of all GM products approved for supply in Australia.
Learn more about the roles of the:
The TGA also recommends that those who intend to use GMOs in gene therapy refer to relevant European Union guidelines:
- Gene therapy product quality aspects in the production of vectors and genetically modified somatic cell
- Quality, preclinical and clinical aspects of gene therapy medicinal products.
Importing GM human health products
To import therapeutic goods into Australia, including GM medicines and gene therapies, they must be:
- approved by the TGA
- listed in the Australian Register of Therapeutic Goods.
Under special circumstances, access to unapproved therapeutic goods may be possible through specific policies.
You may also need authorisation from the OGTR.