Gene Therapy

We are now moving into an exciting new phase in the development of gene therapy as a radically-improved treatment option for FA as we now transition from research into the clinical trials phase after many many years of unrequited promises. The promise that gene therapy holds is to treat bone marrow failure, prevent leukaemia, reduce incidence of head and neck cancer and improve quality of life and survival prospects.

The International Fanconi Anaemia Gene Therapy Working Group is instrumental in bringing together FA experts and those from other sections of the medical community who have an involvement in gene therapy processes, techniques and treatments. The Working Group serves to coordinate potential trials activities globally to accelerate the transition from trials activities to the point where gene therapy becomes the principal treatment option for FA patients.

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