Recent Advances in Nucleic-Acid Based Drugs Development
A special issue of Molecules (ISSN 1420-3049). This special issue belongs to the section "Medicinal Chemistry".
Deadline for manuscript submissions: 31 August 2026 | Viewed by 16
Special Issue Editors
Interests: organic synthesis; synthetic bioactive compounds; bile acid derivatives; modified oligonucleotides
Special Issues, Collections and Topics in MDPI journals
Interests: organic synthesis; drug design; liquid chromatography-mass spectrometry
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Nucleic-acid therapeutics represent a rapidly evolving and promising class of drugs in modern biomedicine. Their unique ability to target specific genetic sequences offers a transformative approach to disease treatment, shifting the paradigm from protein-level intervention to direct modulation of gene expression. This precision enables the development of therapies that act on a single gene or RNA species, offering enhanced specificity and reduced off-target effects compared to conventional drugs.
Recent advances have expanded the repertoire of nucleic acid structures used in therapy, such as aptamers, antisense oligonucleotides, siRNAs, and mRNA-based agents. These molecules can regulate gene expression, inhibit pathogenic RNA, or serve as templates for protein synthesis, making them versatile tools for treating genetic disorders, cancers, viral infections, and neurodegenerative diseases. Chemical innovations (e.g., PS backbones, 2-F/2-OMe, LNA, PMO) extend their half-life and potency, while delivery breakthroughs (e.g., lipid nanoparticles, polymer carriers, and hepatocyte-targeted GalNAc) solve tissue access and lower dosing. Precision, modular design, and scalable manufacturing make nucleic acids attractive for personalized medicine and for rapidly addressing newly validated targets that are considered "undruggable" by small molecules or antibodies.
This Special Issue will include both research articles and original reviews, aiming to highlight the latest developments and innovations in the field of nucleic acids. Contributions may include the synthesis and properties of novel or more efficient nucleic acid-based drugs, new therapeutic targets, innovative applications across various fields, and original technologies to enhance these drugs.
Dr. Daniela Perrone
Dr. Maria Luisa Navacchia
Dr. Elena Marchesi
Guest Editors
Manuscript Submission Information
Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.
Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Molecules is an international peer-reviewed open access semimonthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.
Keywords
- nucleic acids
- oligonucleotide synthesis
- modified nucleic acids
- therapeutic applications
- gene expression regulation
- precision medicine
- undruggable target
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