The therapeutic potential of cell-internalizing aptamers

Area of Science:

• Biotechnology and Molecular Biology
• Drug Delivery Systems
• Nucleic Acid Therapeutics

Background:

• Aptamers, generated via Systematic Evolution of Ligands by Exponential enrichment (SELEX), possess specific binding and structural properties.
• Nucleic acid aptamers present advantages over antibodies, including chemical synthesis, stability, and low immunogenicity.
• Aptamers have found applications in diagnostics, drug development, target validation, and therapeutics over the past two decades.

Purpose of the Study:

• To review recent advancements in utilizing cell-internalizing aptamers as drug delivery vehicles.
• To explore the use of aptamers for targeted delivery, enhancement, and modulation of therapeutic agents.
• To highlight aptamer-mediated, cell-type-specific delivery systems, particularly for siRNA, and their clinical potential.

Main Methods:

• Review of literature on aptamer evolution, properties, and applications in drug delivery.
• Focus on cell-internalizing aptamers targeting cell surface proteins for specific delivery.
• Analysis of aptamer-mediated delivery of various agents: chemical drugs, toxins, siRNAs, and nanoparticle-encapsulated drugs.

Main Results:

• Aptamers can be engineered to target specific cell types for precise drug delivery.
• Cell-internalizing aptamers effectively deliver and modulate the activity of diverse therapeutic payloads.
• Aptamer-mediated siRNA delivery systems show significant promise for cell-type-specific therapeutic applications.

Conclusions:

• Aptamers are versatile tools for targeted drug delivery, offering advantages in specificity and functionality.
• Cell-internalizing aptamers are emerging as powerful escorts for enhancing therapeutic agent delivery and efficacy.
• Further clinical development of aptamer-mediated delivery systems, especially for siRNA, is warranted.