Formulation of Nanoparticulate Systems for Gene and Nucleic Acid Delivery: Gene Therapeutics

Dear Colleagues,

We are pleased to invite you to submit research articles and review articles to this Special Issue on the topic of the formulation of nanoparticulate systems for gene and nucleic acid delivery. Gene therapeutics are very promising to treat difficult-to-treat diseases as they target the cause rather than only the symptoms. However, their formulations and delivery into the body are challenging due to gene complexity regarding the large size, the negative charge and the instability and degradability in the body by nucleases. Therefore, research in this area is needed to overcome the aforementioned challenges and to develop gene therapies in effective, stable and safe forms.

Today, nanoparticulate systems are popular for delivering therapeutics including deoxyribonucleic acid, DNA, and ribonucleic acids, e.g., mRNA. Gene therapy is an expanding field that can treat genetic and acquired diseases. Non-viral gene therapy with liposomes, lipid nanoparticles and polymeric nanoparticles vectors for gene delivery has attracted increasing amounts of interest due to its ability to overcome challenges of viral vectors such as immunogenicity, mutagenicity and oncogenicity. For example, the mRNA-based vaccines (against COVID-19) in lipid nanoparticle forms are recent examples of the successful delivery of a complex mRNA into the body. This reveals the important role of nanoparticles as drug delivery systems. This Special Issue aims to review the most recent advances in research conducted to effectively deliver genes in nanoparticulate drug delivery systems such as lipid nanoparticles, liposomes and polymeric nanoparticles, with a particular focus on the fabrication of nanoparticles and their characterization for inclusion of the gene and nucleic acids.

In this Special Issue we welcome research articles and review articles that cover not only the formulation and stabilization of any type of nanoparticles to include nucleic acids, but also in vitro and in vivo characterization of those complex molecules in nanoparticle forms. Studies exploring novel technologies as microfluidic systems to fabricate nanoparticles are also welcome. Research areas may include (but are not limited to) the following:

• Lipid nanoparticles to deliver mRNA and vaccines;
• Liposomes and gene delivery;
• PLGA and other polymers for nucleic acid delivery;
• Recent advances in nanoparticles that include gene-based therapeutics;
• Gene therapy for rare diseases (non-viral vectors);
• Gene therapy for treating cancer;
• In vitro, in vivo testing of gene therapy.

We look forward to receiving your contributions.

Prof. Dr. Amal Ali Elkordy
Guest Editor

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 2900 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.

• nanoparticles
• liposomes
• lipid nanoparticles
• polymeric nanoparticles
• microfluidic systems
• mRNA
• plasmid DNA
• nucleic acid
• gene delivery