Introduction

Gene therapy for haemophilia

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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?

This resource has 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.  

Date last reviewed: 12 October 2019