Nucleic Acid Therapeutics: From Molecular Mechanisms to Delivery, Manufacturing, and Clinical Translation
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pharmacology".
Deadline for manuscript submissions: 15 October 2026 | Viewed by 127
Editors
Interests: omics; microsystems; cytometry; nanomedicine; cell therapy
Special Issues, Collections and Topics in MDPI journals
Interests: manufacturing; mechanistic modeling; process development; process analytical technology; uncertainty quantification
Special Issue Information
Dear Colleagues,
Nucleic acid therapeutics—including mRNA, siRNA, antisense oligonucleotides, DNA-based therapeutics, and gene-editing platforms—have emerged as powerful modalities for the prevention and treatment of a wide range of diseases. Over the past decade, major advances in molecular design, delivery technologies, and translational development have greatly expanded the promise of this field. However, important challenges remain in achieving efficient delivery, precise targeting, product stability, scalable manufacturing, robust quality control, and safe clinical applications.
Recent progress in lipid nanoparticles, viral vectors, polymer-based carriers, biomaterial-enabled delivery systems, and other emerging platforms has accelerated the development of nucleic acid medicines. At the same time, growing attention has been directed toward the molecular and cellular processes that govern therapeutic outcomes, including uptake, intracellular trafficking, endosomal escape, metabolism, biodistribution, and host immune responses. In parallel, advances in formulation science, analytical characterization, process engineering, and regulatory strategy are playing an increasingly important role in translating these technologies into clinical practice.
This Special Issue aims to provide an inclusive platform for high-quality contributions across the full development pipeline of nucleic acid therapeutics—from basic molecular mechanisms to delivery innovation, manufacturing solutions, and clinical translation. We welcome original research articles, reviews, communications, and perspectives that advance fundamental understanding, enable technological innovation, or address key translational and industrial challenges. Interdisciplinary studies bridging experimental, computational, engineering, and clinical approaches are particularly encouraged.
We are pleased to invite you to contribute to this Special Issue and share your latest work in this rapidly evolving and highly impactful field.
In this Special Issue, original research articles, review papers, and perspectives are welcome. Relevant topics include, but are not limited to:
- Molecular mechanisms underlying nucleic acid therapeutic performance.
- Design and optimization of nucleic acid delivery systems, including lipid nanoparticles, polymers, viral vectors, extracellular vesicles, and hybrid platforms.
- Cellular uptake, intracellular trafficking, endosomal escape, and other biological barriers to delivery.
- Tissue targeting, biodistribution, pharmacokinetics, and therapeutic efficacy.
- Cellular metabolism, immunogenicity, and host responses to nucleic acid therapeutics.
- Formulation development, stability, storage, and dosage form engineering.
- Scalable manufacturing, process development, and quality assurance.
- Analytical methods for characterization, quantification, and batch consistency assessment.
- Preclinical evaluation, safety assessment, and translational studies.
- Regulatory considerations.
- CMC challenges and industrial translation.
- Computational modeling, machine learning, and systems engineering for therapeutic and delivery design.
We look forward to receiving your contributions.
Dr. Zongjie Wang
Guest Editor
Dr. Jie Wang
Guest Editor Assistant
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 250 words) can be sent to the Editorial Office for assessment.
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Keywords
- nucleic acid delivery
- nanoparticle delivery systems
- intracellular trafficking
- endosomal escape
- cellular metabolism
- immunogenicity
- process development
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