Functional Nanomaterials for Gene Therapy

A special issue of Journal of Functional Biomaterials (ISSN 2079-4983). This special issue belongs to the section "Biomaterials for Cancer Therapies".

Deadline for manuscript submissions: 31 October 2026 | Viewed by 2028

Special Issue Editors


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Guest Editor
Department of Bioengineering, University of California, Berkeley, CA, USA
Interests: mRNA delivery; lipid nanoparticles; genome editing technologies; bioconjugation; cancer research

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Guest Editor
UWA Centre for Medical Research, UWA Medical School, Harry Perkins Institute of Medical Research, Perth, Australia
Interests: epigenome manipulation in cancer; CRISPR/Cas9 in immunotherapy; targeted drug delivery for solid tumors

Special Issue Information

Dear Colleagues,

The field of functional nanomaterials for gene therapy is advancing fast, with the need for precise, efficient, and safe delivery vehicles for genetic cargo. Gene therapy holds great potential for the treatment of genetic disorders, cancers, and other complex diseases, but its clinical use is greatly reliant on the development of new nanomaterial-based delivery systems. Functional nanomaterials, including lipid nanoparticles, polymeric carriers, inorganic nanoparticles, and hybrid systems, are pioneering the breach of biological barriers, enhancing targeted delivery, and improving therapeutic efficacy. This Special Issue will highlight recent advances in functional nanomaterials designed for gene therapy applications. We welcome original research and review articles that cover, but are not limited to, the following topics:

  • Design and development of nanomaterials for nucleic acid delivery.
  • Lipid-based nanoparticles (LNPs) for gene therapy.
  • Polymer-based and hybrid nanoparticle systems.
  • Targeted and stimuli-responsive gene delivery platforms.
  • CRISPR/Cas-based genome editing delivery systems.
  • Biocompatibility, toxicity, and immune response of nanocarriers.
  • Translational and clinical advancements in nanomaterial-based gene therapies.

This Special Issue will provide a comprehensive perspective on the current state of the field, bridging fundamental research and clinical applications. By integrating novel material designs with cutting-edge gene therapy strategies, we aim to facilitate the next generation of nanomedicine innovations.

We look forward to your valuable contributions to this exciting Special Issue.

Dr. Rohit Sharma
Dr. Pilar Blancafort
Guest Editors

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Keywords

  • functional nanomaterials
  • gene therapy
  • lipid nanoparticles (LNPs)
  • nucleic acid delivery
  • CRISPR/Cas gene editing
  • biocompatibility and toxicity
  • targeted drug delivery

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Published Papers (1 paper)

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Research

18 pages, 3870 KB  
Article
Nanotherapy Targeting miR-10b Improves Survival in Orthotopic Glioblastoma Models
by Bryan D. Kim, Ming Chen, Sujan K. Mondal, Elizabeth Kenyon, Christiane L. Mallett, Ana deCarvalho, Zdravka Medarova and Anna Moore
J. Funct. Biomater. 2026, 17(1), 15; https://doi.org/10.3390/jfb17010015 - 26 Dec 2025
Cited by 1 | Viewed by 972
Abstract
Glioblastoma (GBM) is the most aggressive primary cancer with poor survival. In the absence of an effective treatment and a high probability of recurrence, new therapeutic approaches are urgently needed. This study focused on targeting microRNA-10b (miR-10b) highly expressed in GBM cells that [...] Read more.
Glioblastoma (GBM) is the most aggressive primary cancer with poor survival. In the absence of an effective treatment and a high probability of recurrence, new therapeutic approaches are urgently needed. This study focused on targeting microRNA-10b (miR-10b) highly expressed in GBM cells that has been identified as one of the key drivers of GBM progression. Inhibiting miR-10b using antisense oligonucleotides (ASOs) has shown promise, but its delivery is challenging due to short circulation half-life, degradation by nucleases, and limited blood–brain barrier (BBB) permeability. To overcome these barriers, we employed a magnetic nanoparticle (MN) platform to deliver anti-miR-10b ASOs (MN-anti-miR10b). In addition to serving as a delivery vehicle, these nanoparticles can be used for monitoring delivery using magnetic resonance imaging (MRI). In therapeutic studies in orthotopic models of GBM presented here we used MN-anti-miR10b as well as TTX-MC138, a clinically tested anti-miR10b nanotherapeutic now in Phase I trials in patients with solid (non-GBM) cancers. Both formulations showed efficient delivery, as demonstrated by imaging and improved survival, leading to target inhibition and increased apoptosis. This approach may offer a novel strategy for delivering therapeutics to GBM and improving patient outcomes in one of the most aggressive and treatment-resistant forms of brain cancer. Full article
(This article belongs to the Special Issue Functional Nanomaterials for Gene Therapy)
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