Background: Research into the molecular and genetic basis of disease is continually expanding. How does the increasing knowledge about the genetic basis of eye diseases contribute to the development of new therapeutic strategies?
Materials and methods: Gene therapy, here defined as the introduction of genetic material into human cells, offers great opportunities. Gene transfer strategies can be used for gene replacement in recessive disease, gene inactivation in dominant disease, expression of "rescue factors" and apoptosis modulators in degenerative disease, "suicide genes" for example in proliferative diseases and expression of immunmodulatory factors in immunological disorders. 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 gene transfer. Adenovirus (AV), adenoassociated virus (AAV), encapsulated adenovirus mini-chromosomes (EAMs), herpes simplex virus (HSV) and lentiviruses are the most frequently used viral vector systems to date. Their advantages 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 growing functional success and may develop into a new therapeutic tool for clinical ophthalmology.