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Recent Advances in RNA Drug Development
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Recent Advances in RNA Drug Development

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: 20 October 2025 | Viewed by 5619

Special Issue Editor

Dr. Hidenori Tani

E-Mail Website
Guest Editor
Department of Health Pharmacy, Yokohama University of Pharmacy, 601 Matano, Totsuka, Yokohama, Japan
Interests: lncRNA function; RNA stability; RNA drug discovery; liquid–liquid phase separation (LLPS); stress response; RNA splicing; machine learning; RNA–protein interaction network
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

RNA therapeutics have emerged as a promising field in drug development, offering innovative approaches to treat various diseases. This Special Issue provides a comprehensive overview of the latest advances and future prospects in RNA drug development, from basic science to clinical applications.

We welcome original research articles and review manuscripts addressing the following topics. (a) RNA therapeutic modalities: Submissions exploring different types of RNA therapeutics, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), aptamers, and CRISPR-Cas9 guide RNAs. Articles discussing their mechanisms of action and therapeutic potential are also of interest. (b) Delivery systems for RNA therapeutics: Highlighting various delivery methods such as lipid nanoparticles, polymeric nanoparticles, conjugation strategies, and viral vectors. Evaluations of their efficacy and limitations are welcome. (c) Clinical applications and challenges: Papers focusing on approved RNA therapies and ongoing trials in areas like neurodegenerative diseases, genetic disorders, cancer, infectious diseases, and cardiovascular conditions. Discussions on challenges in RNA drug development are encouraged. (d) Emerging technologies and regulatory considerations: Submissions exploring novel approaches to overcome current challenges, such as chemical modifications, new delivery platforms, combination therapies, and personalized RNA medicines. Articles addressing regulatory considerations and clinical trials are also of interest.

This Special Issue will serve as a valuable resource for researchers, clinicians, and industry professionals in RNA therapeutics and drug development.

Dr. Hidenori Tani
Guest Editor

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. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. 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

  • RNA therapeutics
  • drug development
  • antisense oligonucleotides
  • siRNA
  • miRNA
  • aptamers
  • CRISPR-Cas9
  • delivery systems
  • clinical applications
  • emerging technologies

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Published Papers (3 papers)

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Review

45 pages, 4152 KiB  
Review
Connecting the Dots: How MicroRNAs Link Asthma and Atherosclerosis
by Răzvan-Ionuț Zimbru, Elena-Larisa Zimbru, Florina-Maria Bojin, Laura Haidar, Minodora Andor, Octavia Oana Harich, Gabriela Tănasie, Carmen Tatu, Diana-Evelyne Mailat, Iulia-Maria Zbîrcea, Bogdan Hirtie, Cristina Uța, Camelia-Felicia Bănărescu and Carmen Panaitescu
Int. J. Mol. Sci. 2025, 26(8), 3570; https://doi.org/10.3390/ijms26083570 - 10 Apr 2025
Viewed by 494
Abstract
Asthma and atherosclerosis are chronic conditions with distinct pathophysiologies, but overlapping inflammatory mechanisms that suggest a potential common regulatory framework. MicroRNAs (miRNAs), small non-coding RNA molecules that modulate gene expression post-transcriptionally, could be key players in linking these disorders. This review outlines how [...] Read more.
Asthma and atherosclerosis are chronic conditions with distinct pathophysiologies, but overlapping inflammatory mechanisms that suggest a potential common regulatory framework. MicroRNAs (miRNAs), small non-coding RNA molecules that modulate gene expression post-transcriptionally, could be key players in linking these disorders. This review outlines how miRNAs contribute to the complex interplay between asthma and atherosclerosis, focusing on key miRNAs involved in inflammatory pathways, immune cell regulation and vascular remodeling. We discuss specific miRNAs, such as miR-155, miR-21 and miR-146a, which have been shown to modulate inflammatory cytokine production and T cell differentiation, impacting respiratory and cardiovascular health. The common miRNAs found in both asthma and atherosclerosis emphasize their role as potential biomarkers, but also as therapeutic targets. Understanding these molecular connections may unlock novel approaches for innovative, integrated treatment strategies that address both conditions and may significantly improve patient outcomes. Further research is needed to explore mechanistic pathways and validate the translational potential of miRNA-based interventions in preclinical and clinical settings. Full article
(This article belongs to the Special Issue Recent Advances in RNA Drug Development)
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44 pages, 4366 KiB  
Review
Harnessing the Potential of Exosomes in Therapeutic Interventions for Brain Disorders
by Lu Bai, Leijie Yu, Mengqiong Ran, Xing Zhong, Meng Sun, Minhao Xu, Yu Wang, Xinlei Yan, Robert J. Lee, Yaqin Tang and Jing Xie
Int. J. Mol. Sci. 2025, 26(6), 2491; https://doi.org/10.3390/ijms26062491 - 11 Mar 2025
Viewed by 866
Abstract
Exosomes, which are nano-sized natural vesicles secreted by cells, are crucial for intercellular communication and interactions, playing a significant role in various physiological and pathological processes. Their characteristics, such as low toxicity and immunogenicity, high biocompatibility, and remarkable drug delivery capabilities—particularly their capacity [...] Read more.
Exosomes, which are nano-sized natural vesicles secreted by cells, are crucial for intercellular communication and interactions, playing a significant role in various physiological and pathological processes. Their characteristics, such as low toxicity and immunogenicity, high biocompatibility, and remarkable drug delivery capabilities—particularly their capacity to traverse the blood–brain barrier—make exosomes highly promising vehicles for drug administration in the treatment of brain disorders. This review provides a comprehensive overview of exosome biogenesis and isolation techniques, strategies for the drug loading and functionalization of exosomes, and exosome-mediated blood–brain barrier penetration mechanisms, with a particular emphasis on recent advances in exosome-based drug delivery for brain disorders. Finally, we address the opportunities and challenges associated with utilizing exosomes as a drug delivery system for the brain, summarizing the barriers to clinical translation and proposing future research directions. Full article
(This article belongs to the Special Issue Recent Advances in RNA Drug Development)
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32 pages, 1618 KiB  
Review
Recent Advances and Prospects in RNA Drug Development
by Hidenori Tani
Int. J. Mol. Sci. 2024, 25(22), 12284; https://doi.org/10.3390/ijms252212284 - 15 Nov 2024
Cited by 7 | Viewed by 4126
Abstract
RNA therapeutics have undergone remarkable evolution since their inception in the late 1970s, revolutionizing medicine by offering new possibilities for treating previously intractable diseases. The field encompasses various modalities, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), and messenger RNAs (mRNAs), [...] Read more.
RNA therapeutics have undergone remarkable evolution since their inception in the late 1970s, revolutionizing medicine by offering new possibilities for treating previously intractable diseases. The field encompasses various modalities, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), and messenger RNAs (mRNAs), each with unique mechanisms and applications. The foundation was laid in 1978 with the discovery that synthetic oligonucleotides could inhibit viral replication, followed by pivotal developments such as RNA interference’s discovery in 1998. The COVID-19 pandemic marked a crucial turning point, demonstrating the potential of mRNA vaccines and accelerating interest in RNA-based approaches. However, significant challenges remain, including stability issues, delivery to target tissues, potential off-target effects, and immunogenicity concerns. Recent advancements in chemical modifications, delivery systems, and the integration of AI technologies are addressing these challenges. The field has seen notable successes, such as approved treatments for spinal muscular atrophy and hereditary transthyretin-mediated amyloidosis. Looking ahead, RNA therapeutics show promise for personalized medicine approaches, particularly in treating genetic disorders and cancer. The continued evolution of this field, driven by technological innovations and deeper understanding of RNA biology, suggests a transformative impact on future medical treatments. The purpose of this review is to provide a comprehensive overview of the evolution, current state, and prospects of RNA therapeutics. Full article
(This article belongs to the Special Issue Recent Advances in RNA Drug Development)
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