ABSTRACT
Both antisense oligonucleotide as well as gene therapy aim at the transfer of genetic material to the target cells for preventing or altering a particular disease process. In gene therapy a good gene is inserted into the cell to repair or replace the faulty gene to produce health-giving proteins, while an oligonucleotide can block the disease-causing protein. With the advances in the identification of the major genes responsible for causing various diseases, an increased focus on the use of therapeutic genes and oligonucleotides has developed, including treatments for ocular diseases. Inherited retinal degeneration is one of the most common causes of blindness in the western world, for which there is no therapy at all. The most researched form of inherited retinal degeneration is retinitis pigmentosa (RP; damage to photoreceptors of the retina by a single abnormal gene) with an occurrence up to 1:3000. A number of important developments have been reported in last few years in identifying genetic defects in RP. Gene and oligonucleotide therapy has been successfully applied in retinal diseases, including the introduction of the antisense oligonucleotide Vitravene1 for cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency syndrome (AIDS) and other immuno suppressed states. Thus, an important milestone in antisense therapy for the eye has been reached. In this chapter we will discuss the princi-
ples of gene, oligonucleotide, and ribozyme therapy with an emphasis on delivery of these drugs to the eye.
