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Aptamers: A Review of Their Chemical Properties and Modifications for Therapeutic Application
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Aptamers are short, single-stranded oligonucleotides that bind to specific target molecules. The shape-forming feature of single-stranded oligonucleotides provides high affinity and excellent specificity toward targets. Hence, aptamers can be used as analogs of antibodies. In December 2004, the US Food and Drug Administration approved the first aptamer-based therapeutic, pegaptanib (Macugen), targeting vascular endothelial growth factor, for the treatment of age-related macular degeneration. Since then, however, no aptamer medication for public health has appeared. During these relatively silent years, many trials and improvements of aptamer therapeutics have been performed, opening multiple novel directions for the therapeutic application of aptamers. This review summarizes the basic characteristics of aptamers and the chemical modifications available for aptamer therapeutics.
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![Figure 1 Overall structure of known aptamer–protein complexes with electrostatic surface potential [5]. The RNA aptamer is represented by a yellow ball-and-stick model. (A) Aptamer–thrombin complex at 1.8 Å resolution [13]. (B) Aptamer–nuclear factor-κB complex at 2.45 Å resolution [11]. (C) Aptamer–MS2 coat protein complex at 2.8 Å resolution [12]. (D) Aptamer–Fc region of human IgG1 (hFc1) complex at 2.15 Å resolution [16]. Blue areas: positively charged; red areas: negatively charged.](/paper-figures/326433949fe441b395fc323372023226/aptamers-a-review-of-their-chemical-properties-and-modifications-for-therapeutic-figure-1.jpg)

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“With the increasing recognition of the vital role played by RNA in transferring cellular information and regulating genes, targeting RNAs has become an exciting opportunity to therapeutically modulate cellular processes linked to previously “undruggable” protein targets.550–552”
From Recent advances in targeting the “undruggable” proteins: from drug discovery to clinical trials (2023) · cites this paper as [552]
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Metadata
- Authors
- Adachi, T., & Nakamura, Y.
- Affiliations
- RIBOMIC Inc., Minato-ku, Tokyo 108-0071, Japan
- Institute of Medical Science, The University of Tokyo (JP)
- Corresponding author
- Adachi, Tatsuo
- Venue
- Molecules
- Publisher
- MDPI AG
- Published
- 2019-11-21
- Volume
- 24
- Issue
- 23
- Pages
- 4229
- ISSN
- 1420-3049 (ISSN-L)
- PMC
- PMC6930564
- OpenAlex
- W2989998401
- Cited by
- 300
- References
- 115
- Citations by year
- 2026: 33 · 2025: 50 · 2024: 59 · 2023: 53 · 2022: 44 · 2021: 42 · 2020: 19
- FWCI
- 2.32
- Type
- journal article
- Language
- en
- Open access
- gold
- dx.doi.org
- Licence
- Locations
- Molecules (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- University of Tokyo
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Advanced biosensing and bioanalysis techniques
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › DNA and Nucleic Acid Chemistry
- Keywords
- aptamers, aptamer therapeutics, chemical modification, oligonucleotide chemistry, pegaptanib, vascular endothelial growth factor, age-related macular degeneration, drug delivery
- MeSH terms
- Animals, Humans, Infections, Neoplasms, Aptamers, Nucleotide, Drug Discovery
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