Ocular gene therapy: introduction to the special issue

Abstract

It has been less than two decades since the publication of the first papers to describe gene transfer to the adult mouse eye. Since then ocular gene therapy has made rapid progress, with the first clinical trials to show clinical benefit occurring in 2008. As well as a wide range of programmes to develop gene therapy for relatively rare disorders such as Leber congenital amaurosis (LCA), there are major attempts to develop gene therapy to treat common ocular conditions such as glaucoma and age-related macular degeneration (AMD). From a handful of academic laboratories, there are now dozens of groups engaged in ocular gene therapy, including small biotechs and major pharmaceutical companies. In the field of gene therapy, the eye has now moved to centre stage. The reason for this increase in interest is perhaps an increasing recognition that the eye is an attractive target for gene therapy.