Gene Therapy for Ocular Disease

The tremendous progress in the past decade in delineating the molecular bases of ocular diseases has been accompanied by the realization that it may be possible to harness this information to generate rational treatment strategies. The concept that treatments might be performed at the genetic level has been a driving force in research involving several blinding ocular diseases. Although there has not, as yet, been a demonstration of cure using gene therapy approaches, proof-of-principle has been established in a number of animal models for a diverse set of ocular diseases.