Over the last few years, huge progress has been made with regard to the understanding of
molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the …

Since long before the first approval of gene therapy for retinal disease, ocular gene therapy
has captured the hopes of patients, clinicians, and scientists alike. Indeed, the retina …

The question of where gene therapy is going has almost as many answers as there are
practitioners in the field. Narrowing our focus to diseases of the eye, this diversity of opinions …

Ocular gene therapy involves the introduction of an exogenous gene product to a host's
cellular and genetic machinery for endogenous production of a desired gene product. The …

The eye is an easily accessible, highly compartmentalised and immune-privileged organ
that offers unique advantages as a gene therapy target. Significant advancements have …

Sight-restoring therapy for the visually impaired and blind is a major unmet medical need.
Ocular gene therapy is a rational choice for restoring vision or preventing the loss of vision …

The maturity in our understanding of the genetics and the pathogenesis of disease in
degenerative retinal disorders has intersected in past years with a novel treatment paradigm …

The purpose of this review is to give the general reader a brief overview of the current state
of the field of non-viral ocular gene therapy. For multiple reasons the eye is an excellent …

Gene therapy to treat ocular disorders is still starting, and current therapies are primarily
experimental, with most human clinical trials still in research state, although beginning to …

As gene therapy begins to produce its first clinical successes, interest in ocular gene transfer
has grown owing to the favorable safety and efficacy characteristics of the eye as a target …