Aptamer Oligonucleotides: Novel Potential Therapeutic Agents in Autoimmune Disease

Li, Weibin; Lan, Xiaopeng

doi:10.1089/nat.2014.0529

Cited by 26 publications

(13 citation statements)

References 54 publications

(60 reference statements)

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“…Other therapeutic targets of aptamer studied so far include thrombin, nucleolin, and prostate-specific membrane antigen [43,53,54]. In addition, aptamers have been used to treat aging-related disorders [55], obesity and diabetes mellitus [56,57], cardiovascular diseases [58], infectious diseases [59,60], blood coagulation [61], bone diseases [62], immunological diseases [63], and cancers [64].…”

Section: Figurementioning

confidence: 99%

Recent Progress and Opportunities for Nucleic Acid Aptamers

Byun

2021

Life

102

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Coined three decades ago, the term aptamer and directed evolution have now reached their maturity. The concept that nucleic acid could modulate the activity of target protein as ligand emerged from basic science studies of viruses. Aptamers are short nucleic acid sequences capable of specific, high-affinity molecular binding, which allow for therapeutic and diagnostic applications. Compared to traditional antibodies, aptamers have several advantages, including small size, flexible structure, good biocompatibility, and low immunogenicity. In vitro selection method is used to isolate aptamers that are specific for a desired target from a randomized oligonucleotide library. The first aptamer drug, Macugen, was approved by FDA in 2004, which was accompanied by many studies and clinical investigations on various targets and diseases. Despite much promise, most aptamers have failed to meet the requisite safety and efficacy standards in human clinical trials. Amid these setbacks, the emergence of novel technologies and recent advances in aptamer and systematic evolution of ligands by exponential enrichment (SELEX) design are fueling hope in this field. The unique properties of aptamer are gaining renewed interest in an era of COVID-19. The binding performance of an aptamer and reproducibility are still the key issues in tackling current hurdles in clinical translation. A thorough analysis of the aptamer binding under varying conditions and the conformational dynamics is warranted. Here, the challenges and opportunities of aptamers are reviewed with recent progress.

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Section: Figurementioning

confidence: 99%

Recent Progress and Opportunities for Nucleic Acid Aptamers

Byun

2021

Life

102

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“…14 Presently, only one aptamer drug (Macugen) has been approved by the FDA, but several others are being evaluated in clinical trials. 15 In addition, aptamers have been used to treat aging-related disorders, 16 obesity and diabetes mellitus, 17,18 cardiovascular diseases, 19 infectious diseases, 20,21 blood coagulation, 22 bone diseases, 23 immunological diseases, 24 and cancers 25 (Figure 2).…”

Section: Use Of Aptamers In Theranosticsmentioning

confidence: 99%

<p>Aptamers as Versatile Ligands for Biomedical and Pharmaceutical Applications</p>

Guan¹,

Zhang²

2020

IJN

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Aptamers are a class of targeting ligands that bind exclusively to biomarkers of interest. Aptamers have been identified as candidates for the construction of various smart systems for therapy, diagnosis, bioimaging, and drug delivery due to their high target affinity and specificity. Aptamers are accounted as chemical antibodies that can be readily linked to drugs, sensors, signal enhancers, or nanocarriers for functionalization. Use of aptamer-guided medications, especially nanomedicines, has resulted in encouraging outcomes compared to those use of aptamer-free counterparts. This article reviews recent advances in the use of aptamers as targeting ligands for various biomedical and pharmaceutical purposes. Special interests focus on aptamer-based theranostics, biosensing, bioimaging, drug potentiation, and targeted drug delivery.

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“…[70,71]. Because aptamers contain some advantages over antibodies and other conventional small-molecule therapeutics, such as high specificity, flexible modification, and low adverse effect, they have been shown as a valuable substitute to protein antibodies [72]. Moreover, the strategies developed to chemically modify backbone can further improve affinity and bioavailability of aptamers [73].…”

Section: Aptamersmentioning

confidence: 99%

Small-molecule Nucleic-acid-based Gene-silencing Strategies

Xu¹,

Yang²

2016

Nucleic Acids - From Basic Aspects to Laboratory Tools

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Gene-targeting strategies based on nucleic acid have opened a new era with the development of potent and effective gene intervention strategies, such as DNAzymes, ribozymes, small interfering RNAs siRNAs , antisense oligonucleotides ASOs , aptamers, decoys, etc. These technologies have been examined in the setting of clinical trials, and several have recently made the successful transition from basic research to clinical trials. This chapter discusses progress made in these technologies, mainly focusing on Dzs and siRNAs, because these are poised to play an integral role in antigene therapies in the future.Keywords: Gene-targeting strategies, DNAzymes, siRNAs, basic research, clinical trials . IntroductionOver the past decade, it is known that the advent of oligonucleotide-based gene inactivation agents have provided potential for these to serve as analytical tools and potential treatments in a range of diseases, including cancer, infections, inflammation, etc. During this time, many genes have been targeted by specifically engineered agents from different classes of smallmolecule nucleic-acid-based drugs in experimental models of disease to probe, dissect, and characterize further the complex processes that underpin molecular signaling. Subsequently, a number of molecules have been examined in the setting of clinical trials, and several have recently made the successful transition from the bench to the clinic, heralding an exciting era of gene-specific treatments. This is particularly important because clear inadequacies in present therapies account for significant morbidity, mortality, and cost. The broad umbrella of gene-silencing therapeutics encompasses a range of agents that include deoxyribozymes DNAzymes, Dzs , ribozymes, siRNAs, ASOs, aptamers, and decoys. This chapter tracks © 2016 The Author(s). Licensee InTech. This chapter is distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. current movements in these technologies, focusing mainly on Dzs and siRNAs, because these are poised to play an integral role in antigene therapies in the future. . DNAzymesAmong the gene-silencing technologies, Breaker and Joyce, in , used an in vitro selection method to identify a special Dz from a random pool of single-stranded DNA to catalyze Pb + -dependent cleavage of an RNA phosphodiester linkage [ ]. Afterward, a number of Dzs were created with the capacity to catalyze many reactions, including the cleavage of DNA or RNA, the modification and ligation of DNA, and the metalation of porphyrin rings. However, because of the low efficiency of RNA cleavage, they are not widely used for biological applications except for -Dz [ ]. The inherent catalytic RNA-cleaving property of Dzs has been used with different mRNA targets as in vitro diagnostic and analytical tools, as well as in vivo therapeutic agents. . . The possible mechanism...

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Aptamer Oligonucleotides: Novel Potential Therapeutic Agents in Autoimmune Disease

Cited by 26 publications

References 54 publications

Recent Progress and Opportunities for Nucleic Acid Aptamers

Recent Progress and Opportunities for Nucleic Acid Aptamers

<p>Aptamers as Versatile Ligands for Biomedical and Pharmaceutical Applications</p>

Small-molecule Nucleic-acid-based Gene-silencing Strategies

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