Aptamers as Innovative Diagnostic and Therapeutic Agents in the Central Nervous System

Vittorio      de Franciscis; Carla   Lucia   Esposito; Silvia      Catuogno; Luciano      Cellai; Laura      Cerchia

Abstract

Aptamers are short non-naturally occurring single stranded DNA or RNA able to bind tightly, due to their specific three-dimensional shapes, to a multitude of targets ranging from small chemical compounds to cells and tissues. Since their first discovery, aptamers became a valuable research tool and show great application to fundamental research, drug selection and clinical diagnosis and therapy. Thanks to their unique characteristics (low size, good affinity for the target, no immunogeneicity, chemical structures that can be easily modified to improve their in vivo applications), aptamers may represent a valid alternative to antibodies particularly for the treatment of neurological disorders that urgently need modalities for drug delivery through the blood brain barrier. Aptamers have excellent potential as reagents for the targeted delivery of active drug substances, either through direct conjugation to the aptamer, or through their encapsulation in aptamer-coated vesicles. We will review here the recent and innovative methods that have been developed and the possible applications of aptamers as inhibitors or tracers in neurological disorders and brain cancer.

Keywords: Alzheimer's disease, aptamers, blood-brain barrier, brain cancer, central nervous system, Parkinson's disease, prion diseases, SELEX