Retinal Gene Therapy

The eye is an excellent target for gene therapy. As a ‘surface’ organ, the eye is easily accessible. Its small size means that correspondingly small treatment doses are required for efficacy. This fact, along with the eye’s compartmentalisation, may limit the potential for systemic effects of intraocular therapies. The eye is also immunologically privileged under normal circumstances, thus limiting both intraocular inflammation and neutralising effects of the immune system on biological treatments. Its normally transparent media facilitates examination and imaging via optical means and, in the case of inherited retinal disease, the fact that it is a paired organ facilitates clinical trial design by providing an inbuilt control.