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Nanomedicine in Gene Therapy and Immunotherapy

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Immunology".

Deadline for manuscript submissions: 20 December 2025 | Viewed by 2236

Special Issue Editor

Department of Ophthalmology and Visual Sciences, Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53705, USA
Interests: nanomedicine; drug delivery; gene delivery; genome editing; immunotherapy

Special Issue Information

Dear Colleagues,

Nanomedicine has emerged as a promising field, offering innovative solutions for targeted gene therapy and immunotherapy as new therapies for various diseases. By harnessing the unique properties of nanomaterials, researchers aim to overcome the limitations of conventional therapies and enhance treatment efficacy and safety. This Special Issue seeks to showcase cutting-edge research and developments in the application of nanomedicine to gene therapy and immunotherapy. We invite contributions that explore the design, synthesis, and characterization of novel nanomaterials for drug delivery, gene delivery, genome editing, and immune modulation. This Special Issue will focus on the design of novel delivery vectors, new applications of nanomedicines, and translational aspects of nanomedicine, including preclinical studies, clinical trials, and regulatory considerations. Potential topics include nanocarriers for targeted gene delivery, non-viral vectors for CRISPR-Cas9 delivery, nanoparticle-mediated immunotherapy, nanomedicine-enabled immunomodulation, and many others. We encourage interdisciplinary collaborations and welcome submissions from diverse fields, including chemistry, materials science, biomedical engineering, biology, immunology, and clinical medicine.

Dr. Ruosen Xie
Guest Editor

Manuscript Submission Information

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Keywords

  • nanomedicine
  • drug delivery
  • gene delivery
  • genome editing
  • immunotherapy

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Published Papers (2 papers)

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Research

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13 pages, 2302 KiB  
Article
Immunotherapy Platform That Conjugates Antigen to Complement C3-Targeted Liposomes Induces a Robust Adaptive Immune Response
by R. G. Barber, Steven Cherry, Sydney Stephens, Kristine Mann, Holly A. Martinson and Max Kullberg
Int. J. Mol. Sci. 2025, 26(11), 4985; https://doi.org/10.3390/ijms26114985 - 22 May 2025
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Abstract
The activation of immunosuppressed antigen-presenting cells (APCs) in the tumor microenvironment is a key goal in modern cancer immunotherapy. Our laboratory utilizes a liposome-based immunotherapy platform that binds endogenous complement to deliver antigen, adjuvant, and therapeutic agents to APCs in vivo. The liposomes [...] Read more.
The activation of immunosuppressed antigen-presenting cells (APCs) in the tumor microenvironment is a key goal in modern cancer immunotherapy. Our laboratory utilizes a liposome-based immunotherapy platform that binds endogenous complement to deliver antigen, adjuvant, and therapeutic agents to APCs in vivo. The liposomes contain external linker groups, which readily bind complement protein C3, and mediate liposomal uptake via complement receptor 3 into APCs. To test the ability of a model antigen to bind to these external linker groups on C3-liposomes and elicit a robust adaptive immune response, we conjugated a modified ovalbumin peptide (OVA-C) to the liposomes and incorporated a toll-like receptor (TLR) 4 agonist, monophosphoryl lipid A (MPLA), in the liposomal membrane. Adaptive immune responses from C57BL/6 mice were analyzed by ELISA and ELISpot. Mice vaccinated with OVA-C liposomes elicited significantly greater humoral and cellular adaptive responses relative to controls. Furthermore, female mice vaccinated with MPLA + OVA-C liposomes produced significantly more IgG antibodies than males vaccinated with the same liposomes. In conclusion, antigen binding on the exterior of C3-liposomes markedly improves antigen loading efficiency and still allows for complement C3-targeted delivery to APCs. These data demonstrate the initiation of a robust cellular and humoral immune response using a new liposomal delivery platform. Full article
(This article belongs to the Special Issue Nanomedicine in Gene Therapy and Immunotherapy)
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Review

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43 pages, 2810 KiB  
Review
Emerging Gene Therapy Based on Nanocarriers: A Promising Therapeutic Alternative for Cardiovascular Diseases and a Novel Strategy in Valvular Heart Disease
by Haoran Yang, Junli Li, Chengxiang Song, Hongde Li, Qiang Luo and Mao Chen
Int. J. Mol. Sci. 2025, 26(4), 1743; https://doi.org/10.3390/ijms26041743 - 18 Feb 2025
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Abstract
Cardiovascular disease remains a leading cause of global mortality, with many unresolved issues in current clinical treatment strategies despite years of extensive research. Due to the great progress in nanotechnology and gene therapy in recent years, the emerging gene therapy based on nanocarriers [...] Read more.
Cardiovascular disease remains a leading cause of global mortality, with many unresolved issues in current clinical treatment strategies despite years of extensive research. Due to the great progress in nanotechnology and gene therapy in recent years, the emerging gene therapy based on nanocarriers has provided a promising therapeutic alternative for cardiovascular diseases. This review outlines the status of nanocarriers as vectors in gene therapy for cardiovascular diseases, including coronary heart disease, pulmonary hypertension, hypertension, and valvular heart disease. It discusses challenges and future prospects, aiming to support emerging clinical treatments. This review is the first to summarize gene therapy using nanocarriers for valvular heart disease, highlighting their potential in targeting challenging tissues. Full article
(This article belongs to the Special Issue Nanomedicine in Gene Therapy and Immunotherapy)
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