In vivo gene transfer into corneal epithelial progenitor cells by viral vectors.

Abstract

Gene therapy is defined as the treatment of diseases by the transfer of new genes into patient’s cells. This therapeutic strategy was originally developed to treat genetic disorders. However, acquired disorders such as cancer and other non-genetic diseases are also important potential targets for gene therapy. The first gene therapy clinical trial was started in 1990, and since then more than 3,000 patients have been treated by gene transfer techniques. Many ocular diseases have been proposed as potential targets for gene therapy. shows the current status of ocular gene therapy research. Various types of vectors containing therapeutic genes or marker genes have been used to transfer genes into ocular target cells. However, it has generally been difficult to achieve efficient and stable gene expression in these target cells. Clinical trials of ocular gene therapy have yet to be conducted.