Oligonucleotide Aptamers for Diagnostics and Therapy

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: 31 March 2026 | Viewed by 447

Special Issue Editors


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Guest Editor
Department of Chemical Sciences, University of Naples Federico II, 80126 Naples, Italy
Interests: non-canonical nucleic acids; nucleic acid theranostics; small molecule–DNA interactions
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Special Issue Information

Dear Colleagues,

In recent decades, oligonucleotide aptamers have emerged as versatile and powerful tools in biomedical research and clinical applications. These short, single-stranded nucleic acids can fold into unique three-dimensional structures able to bind a plethora of different targets—including small molecules, proteins, cells, and even tissues—with high affinity and specificity.

Compared to antibodies, aptamers offer several advantages, such as enhanced chemical stability, low immunogenicity, rapid and cost-effective synthesis, and ease of chemical modification, all of which have significantly expanded their translational potential. As a result, aptamers are being increasingly explored for both diagnostic and therapeutic purposes, such as targeted drug delivery, inhibition of pathogenic proteins, and the development of new treatment strategies for complex diseases.

This Special Issue aims to provide a comprehensive and multidisciplinary overview of the most recent advances in the field of aptamers, highlighting both opportunities and challenges in their application to diagnostics and therapy.

We invite researchers to submit original papers, communications, and reviews regarding oligonucleotide aptamers for diagnostics and therapy to be considered for publication in this Special Issue of Pharmaceutics.

We look forward to hearing from you.

Dr. Claudia Riccardi
Dr. Ettore Napolitano
Guest Editors

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Keywords

  • aptamers
  • oligonucleotides
  • therapy
  • diagnostics
  • targeted drug delivery
  • molecular recognition
  • aptamer engineering
  • protein–aptamer interactions
  • aptamer-based nanotechnology

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Published Papers (1 paper)

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Research

23 pages, 5407 KB  
Article
New Player in the Field of Glioblastoma Therapy: EGFRvIII-Specific Gol1 Aptamer Shows a Great Therapeutic Potential
by Fatima Dzarieva, Svetlana Pavlova, Lika Fab, Dzhirgala Shamadykova, Alexander Revishchin, Anna Alekseeva, Alexey Kopylov, Igor Pronin and Galina Pavlova
Pharmaceutics 2026, 18(3), 299; https://doi.org/10.3390/pharmaceutics18030299 - 27 Feb 2026
Viewed by 148
Abstract
Background: This study aimed to develop a superior aptamer-based therapeutic for targeted glioblastoma intervention by conducting a comparative analysis of two DNA aptamers: the original U2 sequence, selected against glioblastoma cells exhibiting high EGFRvIII expression, and its modified, shortened, and stabilized variant, Gol1. [...] Read more.
Background: This study aimed to develop a superior aptamer-based therapeutic for targeted glioblastoma intervention by conducting a comparative analysis of two DNA aptamers: the original U2 sequence, selected against glioblastoma cells exhibiting high EGFRvIII expression, and its modified, shortened, and stabilized variant, Gol1. Methods: The effects of the investigated aptamers on primary human glioblastoma cells with graded receptor expression levels and on a rat 101/8 glioblastoma tissue model were rigorously studied. Results: The results demonstrated the significant superiority of the stabilized Gol1 aptamer, which exhibited exceptional binding affinity for the EGFRvIII receptor. Pronounced antiproliferative and antimigratory effects against EGFRvIII-positive human tumor cells, ultimately inducing complete cell death. Transcriptomic analysis revealed a sophisticated dual mechanism of action for Gol1: the specific activation of neuronal differentiation genes concurrent with the suppression of key alternative splicing factors. Crucially, in vivo confirmation showed highly selective accumulation of the FAM-labeled Gol1 aptamer exclusively within tumor tissue, with a maximum concentration gradient observed in the invasive border zone and a complete absence of accumulation in intact brain parenchyma. Conclusions: These comprehensive findings confirm that the Gol1 aptamer constitutes a highly promising and versatile platform for developing novel targeted theranostic strategies against glioblastoma, offering a precise approach for both diagnostic imaging and therapeutic intervention. Full article
(This article belongs to the Special Issue Oligonucleotide Aptamers for Diagnostics and Therapy)
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