Gene therapy

No. While gene therapy acts to stop the progression of a disease, it typically does not correct damage that has already occurred.

To illustrate, if a person has a disease leading to significant nerve damage, gene therapy may prevent further damage, reduce pain, and assist in recovery, however the nerves themselves will not necessarily regenerate.

Since gene therapy is only just beginning to be used as a treatment, some of these guidelines are still evolving. As such we encourage you to confirm with your medical specialist if the information you find here or elsewhere matches up with current practice.

Participation in a clinical trial can also depend on a number of factors, such as age, weight, and progression of disease to name just a few. See more information on enrolling in a clinical trial.

Gene therapy is an umbrella term for the different ways we can modify genes as a treatment option.

A common form of gene therapy is gene transfer therapy or gene replacement therapy. Gene transfer therapy is when new genetic material is delivered to certain cells, but this does not usually alter the cells own DNA. Learn more about gene transfer therapy.

Genome editing, or gene editing therapy, is another type of gene therapy. Gene editing therapy is when editing tools change existing genetic material in a cell. Learn more about gene editing therapy.

There are several reasons. It is primarily because gene therapy presents an exciting new possibility for how we may treat disease and while in its infancy we are already seeing some promising results.

This question can become complicated, especially considering there is no answer that fits all contexts. As such we recommend asking your general practitioner (GP) or medical specialist with respect to the therapy that may be chosen and the conditions of that therapy.

In general, for an altered gene to be passed down, the changes to the genome must be made to what is known as the germline (cells that produce eggs or sperm).

The Therapeutic Goods Administration (TGA), and The Office of the Gene Technology Regulator (OGTR) are the official bodies who regulate gene therapy treatments in Australia.

Beyond these regulatory bodies, many other factors can impact how likely gene therapy is investigated as a treatment option. These factors include:

• The nature of the disease
• It's performance in research investigations

When you are asked to give your consent to the procedure, you will be asked to sign documents that acknowledge you are aware of what to expect. This usually happens after many discussions with your doctor. If there is anything you do not understand, please ask questions and do not be afraid to ask for more assistance in understanding.

You can see our gene therapy checklist for a guide on what to ask. Resources such as this are a good place to start your journey towards understanding what is involved with gene therapy, and what questions you may wish to ask your doctor.