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Aptamers: multifunctional molecules for biomedical research

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Abstract

Aptamers are single-stranded oligonucleotides that fold into well-defined three-dimensional shapes, allowing them to bind their targets with high affinity and specificity. They can be generated through an in vitro process called “Systemic Evolution of Ligands by Exponential Enrichment” and applied for specific detection, inhibition, and characterization of various targets like small organic and inorganic molecules, proteins, and whole cells. Aptamers have also been called chemical antibodies because of their synthetic origin and their similar modes of action to antibodies. They exhibit significant advantages over antibodies in terms of their small size, synthetic accessibility, and ability to be chemically modified and thus endowed with new properties. The first generation of aptamer drug “Macugen” was available for public use within 25 years of the discovery of aptamers. With others in the pipeline for clinical trials, this emerging field of medical biotechnology is raising significant interest. However, aptamers pose different problems for their development than for antibodies that need to be addressed to achieve practical applications. It is likely that current developments in aptamer engineering will be the basis for the evolution of improved future bioanalytical and biomedical applications. The present review discusses the development of aptamers for therapeutics, drug delivery, target validation and imaging, and reviews some of the challenges to fully realizing the promise of aptamers in biomedical applications.

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Acknowledgments

Funding from Department of Science and Technology (DST), Govt. of India, and research facility provided by Indian Institute of Science Education and Research (IISER), Pune is gratefully acknowledged.

Disclosure

Jayeeta Banerjee declares no conflict of interests. Marit Nilsen-Hamilton declares ownership of Molecular Express Inc., Ames, which is exploring applications of aptamers for combating specific diseases including cancer.

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  1. Biology Department, Indian Institute of Science Education and Research (IISER), 900 NCL Innovation Park, Dr. Homi Bhabha Road, Pune, 411008, India

    Jayeeta Banerjee

  2. Roy J Carver Department of Biochemistry, Biophysics and Molecular Biology, Iowa State University and Ames Laboratory, Ames, IA, 50011, USA

    Marit Nilsen-Hamilton

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Banerjee, J., Nilsen-Hamilton, M. Aptamers: multifunctional molecules for biomedical research. J Mol Med 91, 1333–1342 (2013). https://doi.org/10.1007/s00109-013-1085-2

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