Gene transfer in ophthalmology

Background: Research into the molecular and genetic basis of disease is con tinually expanding. How does the increasing knowledge about the genetic bas is of eye diseases contribute to the development of new therapeutic strateg ies? Materials and methods: Gene therapy, here defined as the introduction of ge netic material into human cells, offers great opportunities. Gene transfer strategies can be used for gene replacement in recessive disease, gene inac tivation in dominant disease, expression of "rescue factors" and apoptosis modulators in degenerative disease, "suicide genes" for example in prolifer ative diseases and expression of immunmodulatory factors in immunological d isorders. Viral vector systems have been developed to introduce the gene of interest into the target cell. Results: Most of the published strategies include the use of vectors for ge ne transfer. Adenovirus (AV), adenoassociated virus (AAV), encapsulated ade novirus mini-chromosomes (EAMs), herpes simplex virus (HSV) and lentiviruse s are the most frequently used viral vector systems to date. Their advantag es and disadvantages, the in vivo models used for gene transfer in retinal degeneration, and the results obtained to date by different research groups in the field will be reviewed. Conclusions: Gene transfer into ocular tissues has been demonstrated with g rowing functional success and may develop into a new therapeutic tool for c linical ophthalmology.