ABSTRACT
This chapter describes the identification and development of the Macugen aptamer, its cellular effects, pharmacokinetics, and ultimate successes in clinical trials. In addition, future perspectives for ribonucleic acid aptamer-based therapies like Macugen for broadened disease treatment will be discussed. Vascular endothelial growth factor (VEGF) is a member of a growth factor gene family that also includes VEGF-B, VEFG-C, VEGF-D, and placental growth factor. VEGFs are the key signaling proteins involved in both de novo vasculogenesis and angiogenesis from preexisting vasculature. A study measured the vitreous levels of VEGF in eyes of patients with and without proliferative diabetic retinopathy and showed that vitreous VEGF was at significantly higher concentrations in eyes of patients with the disease. The chapter discusses the historical invention and pioneering success of the first therapeutic aptamer Macugen, which, without a doubt, will open the door for many more applications for aptamers in the future.
