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Subspecialties Basic & Translational Research, Retina, Health Economics and Policy

Swimming Against the Nucleotides

Big Ideas

Gene therapies are no longer just hype – they provide tangible benefits to real patients, today. What disease will next fall to this powerful modality? For a clue, look at the biggest gene therapy trial in the world.

Gene therapy has been hyped for many years, in many therapy areas – but for now, it may be ocular disease that benefits most. Regulators have already approved a gene therapy for Leber Congenital Amaurosis (LCA), and similar products are currently being developed for several other diseases of the eye. Some of these are now in clinical trials and attracting commercial interest. Overall, the field is moving very quickly, and it’s an exciting time to be involved, not least in my own field: gene therapies for choroideremia and X-linked retinitis pigmentosa.

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About the Author

Robert MacLaren

Professor of Ophthalmology, University of Oxford, UK

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