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Pharmaceutics
Special Issues
Recent Advances in Nanoparticles for Gene Delivery
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Special Issue "Recent Advances in Nanoparticles for 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 September 2023 | Viewed by 1127

Special Issue Editors

Prof. Dr. Mogie Singh

E-Mail Website
Guest Editor
Nano-Gene and Drug Delivery Group, Department of Biochemistry, Westville Campus, University of KwaZulu-Natal, Provate Bag X54001, Durban 4000, KwaZulu-Natal, South Africa
Interests: nanomedicine; gene/drug delivery; anticancer; gene therapy; nanoparticles; gene expression; gene silencing
Dr. Kaushik Pal

E-Mail Website
Guest Editor
Department of Mechanical and Industrial Engineering, Joint Faculty, Centre of Nano Technology, Indian Institute of Technology, Roorkee 247667, Uttarakhand, India
Interests: nanoparticles; advanced materials; polymer-metal composites; biopolymers; drug delivery; anticancer

Special Issue Information

Dear Colleagues,

Gene therapy has been popular as a strategy to address the shortcomings of many conventional treatments of various diseases. To realise the potential of gene therapy, the delivery of therapeutic genes must be accomplished in a safe and efficient manner. Although viral vectors have been favoured, their safety concerns have been a challenge. Hence, the use of non-viral vehicles such as nanoparticles has generated interest. Nanoparticles have since their inception shown potential for gene delivery, which is still not fully exploited.

This Special Issue aims to collate recent progress in the area of gene delivery using nanoparticles that can promote efficient binding and delivery of specific genes to treatment diseases. We invite articles on the synthesis, characterization, and modification of nanoparticles (lipid-based, organic, inorganic, or carbon-based nanoparticles) that show potential as ideal gene delivery vehicles.  Advances in nanoparticles containing specific targeting ligands or display stimuli-responsive behaviour are of particular interest.

In this Special Issue, original research articles and reviews are welcome. Research areas may include (but are not limited to) the following:

  • Nanoparticles
  • Gene delivery
  • Targeting ligands
  • Nanoparticle functionalization
  • Nanoparticle characterization

We look forward to receiving your contributions.

Prof. Dr. Mogie Singh
Dr. Kaushik Pal
Guest Editors

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. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). 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

  • nanoparticles
  • gene delivery
  • targeting ligands
  • nanoparticle functionalization
  • nanoparticle characterization

Published Papers (1 paper)

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Research

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Article
Production, Characterization, and Assessment of Permanently Cationic and Ionizable Lipid Nanoparticles for Use in the Delivery of Self-Amplifying RNA Vaccines
by
Dylan Kairuz
,
Nazia Samudh
,
Abdullah Ely
,
Patrick Arbuthnot
and
Kristie Bloom
Pharmaceutics 2023, 15(4), 1173; https://doi.org/10.3390/pharmaceutics15041173 - 07 Apr 2023
Viewed by 942
Abstract
Africa bears the highest burden of infectious diseases, yet the continent is heavily reliant on First World countries for the development and supply of life-saving vaccines. The COVID-19 pandemic was a stark reminder of Africa’s vaccine dependence and since then great interest has [...] Read more.
Africa bears the highest burden of infectious diseases, yet the continent is heavily reliant on First World countries for the development and supply of life-saving vaccines. The COVID-19 pandemic was a stark reminder of Africa’s vaccine dependence and since then great interest has been generated in establishing mRNA vaccine manufacturing capabilities on the African continent. Herein, we explore alphavirus-based self-amplifying RNAs (saRNAs) delivered by lipid nanoparticles (LNPs) as an alternative to the conventional mRNA vaccine platform. The approach is intended to produce dose-sparing vaccines which could assist resource-constrained countries to achieve vaccine independence. Protocols to synthesize high-quality saRNAs were optimized and in vitro expression of reporter proteins encoded by saRNAs was achieved at low doses and observed for an extended period. Permanently cationic or ionizable LNPs (cLNPs and iLNPs, respectively) were successfully produced, incorporating saRNAs either exteriorly (saRNA-Ext-LNPs) or interiorly (saRNA-Int-LNPs). DOTAP and DOTMA saRNA-Ext-cLNPs performed best and were generally below 200 nm with good PDIs (<0.3). DOTAP and DDA saRNA-Int-cLNPs performed optimally, allowing for saRNA amplification. These were slightly larger, with higher PDIs as a result of the method used, which will require further optimization. In both cases, the N:P ratio and lipid molar ratio had a distinct effect on saRNA expression kinetics, and RNA was encapsulated at high percentages of >90%. These LNPs allow the delivery of saRNA with no significant toxicity. The optimization of saRNA production and identification of potential LNP candidates will facilitate saRNA vaccine and therapeutic development. The dose-sparing properties, versatility, and manufacturing simplicity of the saRNA platform will facilitate a rapid response to future pandemics. Full article
(This article belongs to the Special Issue Recent Advances in Nanoparticles for Gene Delivery)
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