chapter  21
16 Pages

Potential Therapeutic Applications of Antisense Oligonucleotides in Ophthalmology

WithLisa R. Grillone, Scott P. Henry

In the past several decades tremendous strides have been made toward understanding the molecular basis of various ocular diseases. Modern day ocular pharmacology provides the basis for utilization of rational drug design targeted toward molecular mechanisms in a plethora of ocular conditions with the ultimate goal of developing effective therapeutics [1]. The application of antisense oligonucleotide (ASO) therapeutics for the treatment of ocular diseases and conditions has tremendous opportunity to add treatment option in ophthalmology. This class of pharmacologic agent has several advantages that exemplify rational drug design principles. There is the potential to target a vast array of genes that specifically inhibit cellular processes. The use of ASOs to treat ocular diseases is limited only by our knowledge of the underlying molecular pharmacology. With advancements in the understanding of the molecular basis for ocular diseases, spawned by new

animal and cell-culture models that mimic specific diseases, oligonucleotide therapeutics are well positioned to take advantage of new opportunities in ophthalmology and to address unmet needs.