Abstract
Aptamers are single-stranded nucleic acids that specifically recognize and bind tightly to their cognate targets due to their stable three-dimensional structure. Nucleic acid aptamers have been developed for various applications, including diagnostics, molecular imaging, biomarker discovery, target validation, therapeutics, and drug delivery. Due to their high specificity and binding affinity, aptamers directly block or interrupt the functions of target proteins making them promising therapeutic agents for the treatment of human maladies. Additionally, aptamers that bind to cell surface proteins are well suited for the targeted delivery of other therapeutics, such as conjugated small interfering RNAs (siRNA) that induce RNA interference (RNAi). Thus, aptamer-siRNA chimeras may offer dual-functions, in which the aptamer inhibits a receptor function, while the siRNA internalizes into the cell to target a specific mRNA. This review focuses on the current progress and therapeutic potential of RNA aptamers, including the use of cell-internalizing aptamers as cell-type specific delivery vehicles for targeted RNAi. In particular, we discuss emerging aptamer-based therapeutics that provide unique clinical opportunities for the treatment various cancers and neurological diseases.
Highlights
Aptamers are oligonucleotides selected in vitro to bind target molecules with high affinity and specificity due to their stable three-dimensional shapes
This review focuses on the current progress and therapeutic potential of RNA aptamers, including the use of cell-internalizing aptamers as cell-type specific delivery vehicles for targeted RNA interference (RNAi)
Compared to the antibody drugs, appropriate chemical modifications of therapeutic aptamers can be introduced in the solid-phase synthesis to enhance their bio-stability and the pharmacodynamic (PD) and PK
Summary
Aptamers are oligonucleotides selected in vitro to bind target molecules with high affinity and specificity due to their stable three-dimensional shapes. Cell-based SELEX involves selecting for aptamers that bind to an expressed protein and ensures that the aptamer targets the protein in its in vivo conformation. While antibodies often elicit strong immune responses resulting in a loss of efficacy, immunogenicity is limited by aptamers due to chemical modifications on the aptamers. Due to their small size, aptamers have improved transport and tissue penetration than antibodies. The modifications keep hairpin loops and other single-stranded structures intact to maintain the binding affinity of the aptamer. An increase in stability enhances the binding affinity www.frontiersin.org
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