gms | German Medical Science

The genetic basis of inherited retinal dystrophies suggests that gene therapy is a logical therapeutic approach. It may be used to either replace or reduce the expression of defective genes. Safety and potential effectiveness have been suggested for specific disease entities (Leber’s congenital amaurosis) in first proof-of-principal clinical trials. In contrast to pharmacological treatment gene therapy has the advantage of being able to express a protein within specific cell populations and is a potentially definitive therapy. Besides replacing deficient genes in inherited diseases, additional strategies that might broaden the application of retinal gene therapy are also being developed. These include the permanent expression of neuroprotective substances or photosensitive molecules (so-called optogenetics). This overview presents an overview on current clinical trials and potential problems of retinal gene therapy.