Prospects for gene therapy in corneal disease.

Transfer of cDNA to corneal cells has been accomplished using viral and nonviral vectors. Studies examining the feasibility and optimal methods for vector-mediated gene transfer to the cornea have, as in other tissues, been performed using histochemical or fluorescent marker genes. These have used corneal cells or cell lines in vitro, and whole corneas maintained in ex vivo culture. Gene-based interventions have been examined in specific corneal disorders such as allograft rejection, postexcimer laser scarring, and herpes simplex keratitis using experimental models. As the feasibility of genetic modification of corneal cells has been successfully demonstrated, there is great potential for gene therapy vectors in the treatment of human corneal disease. Continued improvements in vectors for gene transfer will improve the efficacy and safety of gene therapy. In addition to use of cDNA transfer as an alternative to drug or protein treatments in acquired corneal disorders, our expanding knowledge of the genetic basis of inherited corneal disorders will ultimately lead to the development of specific and effective gene therapies in this category of diseases.