Nanoparticle-Based Gene Delivery

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

Deadline for manuscript submissions: 30 June 2025 | Viewed by 967

Special Issue Editors


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Guest Editor
School of Pharmacy, Changzhou University, Changzhou 213164, China
Interests: drug delivery; gene delivery; nanomedicine; biomaterials
Special Issues, Collections and Topics in MDPI journals

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Guest Editor
School of Medicine, Pharmacy and Biomedical Sciences, University of Portsmouth, Portsmouth PO1 2DT, UK
Interests: drug delivery; gene delivery; nanomedicine; cancer research; biomaterials
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Gene therapy involves the introduction of exogenous genetic material into the cells in order to correct a specific inherited or acquired pathological condition. However, efficient delivery of the nucleic acids to the target cells is hampered by a number of extracellular and intracellular barriers which necessitates the use of a suitable gene delivery system. Over the last two decades, there has been a rapid increase in the development of gene therapy products and several new gene therapies have found their way to the market and/or received FDA approval such as Mipomersen (Kynamro®), Inotersen (Tegsedi®), patisiran (Onpattro®) and the mRNA based COVID and cancer vaccines. The fast growth of gene therapies is in part due to the introduction of various types of non-viral nanoparticle-based gene delivery systems most notably lipid-based and polymer-based systems. Compared to viral vectors, the nanoparticle based gene vectors are more versatile, safer (less immunogenic), easier to manufacture and scale up, and have much more potential for functionalization and targeting.  

The focus of this issue of Pharmaceutics is novel nanoparticle-based gene delivery systems for highly efficient delivery of nucleic acid therapeutics to different organs and tissues. The scope of the issue covers every kind of nanoparticles made of polymers, lipids, peptides, proteins, metals, and hybrid systems for delivery of all kinds of therapeutic nucleic acids including pDNA, mRNA and oligonucleotides (ASOs, SSOs, siRNA, shRNA, miRNA).

Prof. Dr. Xiubo Zhao
Dr. Roja Hadianamrei
Guest Editors

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Keywords

  • nanaoparticles
  • gene delivery
  • nucleic acids
  • DNA
  • RNA
  • gene therapy

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Published Papers (1 paper)

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Review

22 pages, 2643 KiB  
Review
Immune Modulation with Oral DNA/RNA Nanoparticles
by Ulpan Kart, Aigul Raimbekova, Sergey Yegorov and Gonzalo Hortelano
Pharmaceutics 2025, 17(5), 609; https://doi.org/10.3390/pharmaceutics17050609 - 4 May 2025
Viewed by 670
Abstract
The oral delivery of DNA/RNA nanoparticles represents a transformative approach in immunotherapy and vaccine development. These nanoparticles enable targeted immune modulation by delivering genetic material to specific cells in the gut-associated immune system, triggering both mucosal and systemic immune responses. Unlike parenteral administration, [...] Read more.
The oral delivery of DNA/RNA nanoparticles represents a transformative approach in immunotherapy and vaccine development. These nanoparticles enable targeted immune modulation by delivering genetic material to specific cells in the gut-associated immune system, triggering both mucosal and systemic immune responses. Unlike parenteral administration, the oral route offers a unique immunological environment that supports both tolerance and activation, depending on the formulation design. This review explores the underlying mechanisms of immune modulation by DNA/RNA nanoparticles, their design and delivery strategies, and recent advances in their application. Emphasis is placed on strategies to overcome physiological barriers such as acidic pH, enzymatic degradation, mucus entrapment, and epithelial tight junctions. Special attention is given to the role of gut-associated lymphoid tissue in mediating immune responses and the therapeutic potential of these systems in oral vaccine platforms, food allergies, autoimmune diseases, and chronic inflammation. Despite challenges, recent advances in nanoparticle formulation support the translation of these technologies into clinical applications for both therapeutic immunomodulation and vaccination. Full article
(This article belongs to the Special Issue Nanoparticle-Based Gene Delivery)
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