Gene therapy for inherited retinal degeneration

Research into the molecular and genetic basis of disease is continually expanding and improving the prospects for rational treatments. Of these, gene therapy (here defined as the introduction of genetic material into human cells) may ultimately oVer the greatest scope. Of the inherited forms of retinal degeneration, retinitis pigmentosa (RP) is the best characterised (see Bird 2 for review). Seven diVerent non-syndromic RP genes have been identified to date, five of which However, photoreceptors and retinal pigment epithelial (RPE) cells are in close proximity and are interdependent. Defects in genes expressed in the RPE may also result in retinal degeneration. Delivery to these tissues of either normal copies of the defective genes or genes which enhance cell survival may arrest the degenerative process and thus preserve vision.