Nanomedicines for Nucleic Acid Delivery

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Nanomedicine and Nanotechnology".

Deadline for manuscript submissions: closed (30 September 2024) | Viewed by 3341

Special Issue Editors


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Guest Editor
Nanotechnology and Gene Therapy Group (PharmaNanoGene), Campus Alava, Universidad del Pais Vasco—Euskal Herriko Unibertsitatea, Vitoria-Gasteiz, Spain
Interests: gene therapy; controlled drug delivery; retinal gene therapy; nonviral vectors; lipid nanoparticles; ocular drug delivery; bioavailability; pharmacokinetics; formulations; nanoparticles drug delivery; pharmacology; nanomedicine; nanotechnology; bioequivalence studies; messenger RNA; therapeutic nucleic acids; lysosomal storage diseases
Special Issues, Collections and Topics in MDPI journals

E-Mail Website
Guest Editor
Nanotechnology and Gene Therapy Group (PharmaNanoGene), Campus Alava, Universidad del Pais Vasco—Euskal Herriko Unibertsitatea, Vitoria-Gasteiz, Spain
Interests: pharmaceutics and pharmaceutical technology; controlled drug delivery; drug formulation development; nanotechnology in drug delivery; pharmaceutical research and development; ocular drug delivery; novel drug delivery systems; gene therapy; retinal gene therapy; nonviral vectors; lipid nanoparticles; messenger RNA; therapeutic nucleic acids; lysosomal storage diseases
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Gene therapy medicinal products are making great progress, leading the way in the development of advanced therapies. Diverse molecules are being used to address the development of this kind of medicines. DNA and messenger RNA (mRNA) induce protein expression, whereas small interfering RNA (siRNA), microRNA, oligonucleotides or aptamers provide posttranslational gene silencing. Molecular scissor and gene editing approaches, such as clustered regularly interspaced short palindromic repeats (CRISPR)-associated nuclease Cas9 (CRISPR/Cas9), are also being developed.

Thanks to the first messenger RNA-based vaccines marketed for COVID-19, nucleic acid-based medicines have recently taken a huge step toward the clinic. Nevertheless, the clinical application of nucleic acids is mostly limited by their instability and their difficulty to pass through the cellular membrane, due to the anionic nature of nucleic acids. The formulation of nucleic acids into nanosystems or vectors tailored to the type of nucleic acid and the therapeutic target is of major importance for advancing in the development of gene therapy.

This Special Issue aims to highlight the current state and future perspectives in the research area of nanomedicines for nucleic acid delivery. It will be focused on innovative delivery systems for plasmid DNA, mRNA, microRNA, aptamers, oligonucleotides, siRNA or gene editing strategies, including the study of their biodistribution at intracellular level, and the evaluation of their efficacy in vivo, ex vivo, and in vitro.

Prof. Dr. María Ángeles Solinís
Prof. Dr. Ana Del Pozo-Rodríguez
Guest Editors

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Keywords

  • nanomedicine
  • nanoparticles
  • gene therapy
  • DNA
  • messenger RNA
  • microRNA
  • oligonucleotide
  • siRNA
  • CRISPR/Cas9
  • intracellular biodistribution

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Published Papers (1 paper)

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Review

20 pages, 2095 KiB  
Review
Smart Nanocarriers for the Targeted Delivery of Therapeutic Nucleic Acid for Cancer Immunotherapy
by Abu Baker, Jochen Lorch, David VanderWeele and Bin Zhang
Pharmaceutics 2023, 15(6), 1743; https://doi.org/10.3390/pharmaceutics15061743 - 15 Jun 2023
Cited by 4 | Viewed by 2591
Abstract
A wide variety of therapeutic approaches and technologies for delivering therapeutic agents have been investigated for treating cancer. Recently, immunotherapy has achieved success in cancer treatment. Successful clinical results of immunotherapeutic approaches for cancer treatment were led by antibodies targeting immune checkpoints, and [...] Read more.
A wide variety of therapeutic approaches and technologies for delivering therapeutic agents have been investigated for treating cancer. Recently, immunotherapy has achieved success in cancer treatment. Successful clinical results of immunotherapeutic approaches for cancer treatment were led by antibodies targeting immune checkpoints, and many have advanced through clinical trials and obtained FDA approval. A major opportunity remains for the development of nucleic acid technology for cancer immunotherapy in the form of cancer vaccines, adoptive T-cell therapies, and gene regulation. However, these therapeutic approaches face many challenges related to their delivery to target cells, including their in vivo decay, the limited uptake by target cells, the requirements for nuclear penetration (in some cases), and the damage caused to healthy cells. These barriers can be avoided and resolved by utilizing advanced smart nanocarriers (e.g., lipids, polymers, spherical nucleic acids, metallic nanoparticles) that enable the efficient and selective delivery of nucleic acids to the target cells and/or tissues. Here, we review studies that have developed nanoparticle-mediated cancer immunotherapy as a technology for cancer patients. Moreover, we also investigate the crosstalk between the function of nucleic acid therapeutics in cancer immunotherapy, and we discuss how nanoparticles can be functionalized and designed to target the delivery and thus improve the efficacy, toxicity, and stability of these therapeutics. Full article
(This article belongs to the Special Issue Nanomedicines for Nucleic Acid Delivery)
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