A path towards restoration of vision using ocular gene therapy: An ophthalmic review

The eye has unique advantages as a target organ for gene therapy of both inherited and acquired ocular disorders and offers a valuable model system for Ocular gene therapy which has made significant advances due to improvements in vector delivery methods. Gene therapy for ocular disease has been under investigation just over 18 years and the important advances include the availability of viral and non-viral vectors that are able to efficiently transduce various ocular cell types, the use of various intraocular delivery routes that allow sustained level of gene transfer in animals. Recently, the first human gene therapy trials for retinal degeneration were undertaken with encouraging preliminary safety and efficacy findings. Although much of the work in ocular gene therapy has involved retinal applications, significant progress has also been seen in other aspects of ophthalmology. The application of gene therapy is still developing, and requires further efforts before it is brought to the clinical level. Development of a successful strategy for gene therapy depends on several factors including the choice of the disease, molecular genetic basis, and mechanism capable to deliver the desired gene to the therapeutic site and finally the expression of the therapeutic gene in the appropriate cells has to be ensued. These Ocular studies have established the foundation for methodology of ocular gene transfer which has the potential to become a valuable therapeutic strategy for various untreatable blinding diseases.