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Advances in our understanding of the molecular pathogenesis of retinal degenerative disorders have led in the past 25 years to the development of a new treatment modality for several forms of retinal diseases, in which gene-based therapies may delay, prevent and even reverse blindness. To enable a full overview of this field, we start this chapter by describing the general principle of gene therapy and the properties of the retina that make it amenable to gene therapy. We then discuss current clinical trials – highlighting the growing application of retinal gene therapy – and the areas that require further effort before retinal gene therapy is applied to new and more complex therapeutic indications.

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