In this section
- Gene therapy for haemophilia
- Types of gene therapy
- Genes
- How gene therapy works
- Having gene therapy
- Clinical trials for gene therapy
- Gene therapy FAQs
It is an exciting time in gene therapy for haemophilia. For a long time gene therapy has been hailed as a potential ‘cure’ for haemophilia, but the expectation was that viable treatment was many years away in the future. In recent years international experimental gene therapy studies have begun to demonstrate successful results in people with haemophilia and are now conducting advanced studies in larger groups. Other experimental gene therapy trials are also commencing. What does this mean for people with bleeding disorders in Australia?
These resources have been developed to answer the questions from Australian bleeding disorders community members about gene therapy and its relevance to them now and in the future.
Gene therapy for haemophilia (booklet)
Information for people with haemophilia about gene therapy and how it works in haemophilia. This booklet explains the types of gene therapy that are being trialled in haemophilia at present, who can have gene therapy, what the process involves for a person having gene therapy, and how effective it is, safety and side-effects. It uses easy to read language and diagrams to explain how gene therapy for haemophilia works in the body. It also covers FAQs from the community. (October 2019)
Gene therapy for haemophilia (fact sheet)
A fact sheet on gene therapy for people with haemophilia, their families and carers. It explains what gene therapy is, the types of gene therapy in haemophilia and how they work; who is eligible for gene therapy; benefits, risks and side-effects; what the treatment process is and how your Haemophilia Treatment Centre can help with advice and support. It also answers FAQs. Includes a diagram on how gene therapy works and is given. (October 2025)