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Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies

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A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: closed (10 November 2021) | Viewed by 59716

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Prof. Dr. Satoshi Uchida

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Medical Chemistry, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kyoto, Japan
Interests: mRNA therapeutics; gene therapy; nanomedicine; RNA nanotechnology
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

The delivery of plasmid DNA (pDNA) and messenger RNA (mRNA) is garnering growing attention as the next generation of therapeutics for intractable diseases, including genetic diseases and cancers. Vaccines against pandemic diseases and cancers are also promising targets of this approach. Indeed, several clinical trials are in progress. Concurrently, basic studies are vigorously performed to improve gene introduction efficiency and minimize toxicity concerns of the delivery systems, by developing novel delivery carriers of pDNA and mRNA and applying them for therapeutic studies in animal models. These efforts are valuable for overcoming the current limitations of non-viral gene therapy to achieve the widespread application of pDNA and mRNA therapeutics in the future. This Special Issue welcomes your contribution in these fields at various stages, from the development of delivery systems to the therapeutic application of the systems, in the form of communications, original articles and reviews.

Prof. Dr. Satoshi Uchida
Guest Editor

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Keywords

plasmid DNA
messenger RNA
lipid nanoparticle
polyplex
physical gene delivery
gene therapy
vaccine
cancer
genetic disease

Published Papers (14 papers)

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Editorial

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4 pages, 180 KiB

Open AccessEditorial

Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies

by Satoshi Uchida

Pharmaceutics 2022, 14(4), 810; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics14040810 - 07 Apr 2022

Cited by 4 | Viewed by 3055

Abstract

The vast potential of non-viral delivery systems of messenger RNA (mRNA) and plasmid DNA (pDNA) has been demonstrated in the vaccines against coronavirus disease 2019 (COVID-19) [...] Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

Research

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21 pages, 3070 KiB

Open AccessArticle

Co-Delivery of mRNA and pDNA Using Thermally Stabilized Coacervate-Based Core-Shell Nanosystems

by Sarah S. Nasr, Sangeun Lee, Durairaj Thiyagarajan, Annette Boese, Brigitta Loretz and Claus-Michael Lehr

Pharmaceutics 2021, 13(11), 1924; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13111924 - 13 Nov 2021

Cited by 11 | Viewed by 3679

Abstract

Co-delivery of different species of protein-encoding polynucleotides, e.g., messenger RNA (mRNA) and plasmid DNA (pDNA), using the same nanocarrier is an interesting topic that remains scarcely researched in the field of nucleic acid delivery. The current study hence aims to explore the possibility of the simultaneous delivery of mRNA (mCherry) and pDNA (pAmCyan) using a single nanocarrier. The latter is based on gelatin type A, a biocompatible, and biodegradable biopolymer of broad pharmaceutical application. A core-shell nanostructure is designed with a thermally stabilized gelatin–pDNA coacervate in its center. Thermal stabilization enhances the core’s colloidal stability and pDNA shielding effect against nucleases as confirmed by nanoparticle tracking analysis and gel electrophoresis, respectively. The stabilized, pDNA-loaded core is coated with the cationic peptide protamine sulfate to enable additional surface-loading with mRNA. The dual-loaded core-shell system transfects murine dendritic cell line DC2.4 with both fluorescent reporter mRNA and pDNA simultaneously, showing a transfection efficiency of 61.4 ± 21.6% for mRNA and 37.6 ± 19.45% for pDNA, 48 h post-treatment, whereas established commercial, experimental, and clinical transfection reagents fail. Hence, the unique co-transfectional capacity and the negligible cytotoxicity of the reported system may hold prospects for vaccination among other downstream applications. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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12 pages, 2927 KiB

Open AccessArticle

mRNA-Based Anti-TCR CDR3 Tumour Vaccine for T-Cell Lymphoma

by Marina Tusup, Severin Läuchli, Natalia Teresa Jarzebska, Lars E. French, Yun-Tsan Chang, Maya Vonow-Eisenring, Andreas Su, Thomas M. Kündig, Emmanuella Guenova and Steve Pascolo

Pharmaceutics 2021, 13(7), 1040; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13071040 - 07 Jul 2021

Cited by 8 | Viewed by 4299

Abstract

Efficient vaccination can be achieved by injections of in vitro transcribed mRNA (ivt mRNA) coding for antigens. This vaccine format is particularly versatile and allows the production of individualised vaccines conferring, T-cell immunity against specific cancer mutations. The CDR3 hypervariable regions of immune receptors (T-cell receptor, TCR or B-cell receptor, BCR) in the context of T- or B-cell leukaemia or lymphoma are targetable and specific sequences, similar to cancer mutations. We evaluated the functionality of an mRNA-based vaccine designed to trigger immunity against TCR CDR3 regions in an EL4 T-lymphoma cell line-derived murine in vivo model. Vaccination against the hypervariable TCR regions proved to be a feasible approach and allowed for protection against T-lymphoma, even though immune escape in terms of TCR downregulation paralleled the therapeutic effect. However, analysis of human cutaneous T-cell lymphoma samples indicated that, as is the case in B-lymphomas, the clonotypic receptor may be a driver mutation and is not downregulated upon treatment. Thus, vaccination against TCR CDR3 regions using customised ivt mRNA is a promising immunotherapy method to be explored for the treatment of patients with T-cell lymphomas. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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10 pages, 1513 KiB

Open AccessCommunication

PEGylation of mRNA by Hybridization of Complementary PEG-RNA Oligonucleotides Stabilizes mRNA without Using Cationic Materials

by Naoto Yoshinaga, Mitsuru Naito, Yoshihiro Tachihara, Eger Boonstra, Kensuke Osada, Horacio Cabral and Satoshi Uchida

Pharmaceutics 2021, 13(6), 800; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13060800 - 27 May 2021

Cited by 11 | Viewed by 4073

Abstract

Messenger RNA (mRNA) delivery strategies are required to protect biologically fragile mRNA from ribonuclease (RNase) attacks to achieve efficient therapeutic protein expression. To tackle this issue, most mRNA delivery systems have used cationic components, which form electrostatically driven complexes with mRNA and shield encapsulated mRNA strands. However, cationic materials interact with anionic biomacromolecules in physiological environments, which leads to unspecific reactions and toxicities. To circumvent this issue of cation-based approaches, herein, we propose a cation-free delivery strategy by hybridization of PEGylated RNA oligonucleotides with mRNA. The PEG strands on the mRNA sterically and electrostatically shielded the mRNA, improving mRNA nuclease stability 15-fold after serum incubation compared with unhybridized mRNA. Eventually, the PEGylated mRNA induced nearly 20-fold higher efficiency of reporter protein expression than unhybridized mRNA in cultured cells. This study provides a platform to establish a safe and efficient cation-free mRNA delivery system. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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12 pages, 1388 KiB

Open AccessArticle

α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice

by Julen Rodríguez-Castejón, Ana Alarcia-Lacalle, Itziar Gómez-Aguado, Mónica Vicente-Pascual, María Ángeles Solinís Aspiazu, Ana del Pozo-Rodríguez and Alicia Rodríguez-Gascón

Pharmaceutics 2021, 13(6), 771; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13060771 - 21 May 2021

Cited by 11 | Viewed by 3543

Abstract

Fabry disease (FD) is a monogenic X-linked lysosomal storage disorder caused by a deficiency in the lysosomal enzyme α-Galactosidase A (α-Gal A). It is a good candidate to be treated with gene therapy, in which moderately low levels of enzyme activity should be sufficient for clinical efficacy. In the present work we have evaluated the efficacy of a non-viral vector based on solid lipid nanoparticles (SLN) to increase α-Gal A activity in an FD mouse model after intravenous administration. The SLN-based vector incremented α-Gal A activity to about 10%, 15%, 20% and 14% of the levels of the wild-type in liver, spleen, heart and kidney, respectively. In addition, the SLN-based vector significantly increased α-Gal A activity with respect to the naked pDNA used as a control in plasma, heart and kidney. The administration of a dose per week for three weeks was more effective than a single-dose administration. Administration of the SLN-based vector did not increase liver transaminases, indicative of a lack of toxicity. Additional studies are necessary to optimize the efficacy of the system; however, these results reinforce the potential of lipid-based nanocarriers to treat FD by gene therapy. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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20 pages, 4548 KiB

Open AccessArticle

Synthesis and Characterization of Mannosylated Formulations to Deliver a Minicircle DNA Vaccine

by Ana Sofia Serra, Dalinda Eusébio, Ana Raquel Neves, Tânia Albuquerque, Himanshu Bhatt, Swati Biswas, Diana Costa and Ângela Sousa

Pharmaceutics 2021, 13(5), 673; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13050673 - 07 May 2021

Cited by 9 | Viewed by 2321

Abstract

DNA vaccines still represent an emergent area of research, giving rise to continuous progress towards several biomedicine demands. The formulation of delivery systems to specifically target mannose receptors, which are overexpressed on antigen presenting cells (APCs), is considered a suitable strategy to improve the DNA vaccine immunogenicity. The present study developed binary and ternary carriers, based on polyethylenimine (PEI), octa-arginine peptide (R8), and mannose ligands, to specifically deliver a minicircle DNA (mcDNA) vaccine to APCs. Systems were prepared at various nitrogen to phosphate group (N/P) ratios and characterized in terms of their morphology, size, surface charge, and complexation capacity. In vitro studies were conducted to assess the biocompatibility, cell internalization ability, and gene expression of formulated carriers. The high charge density and condensing capacity of both PEI and R8 enhance the interaction with the mcDNA, leading to the formation of smaller particles. The addition of PEI polymer to the R8-mannose/mcDNA binary system reduces the size and increases the zeta potential and system stability. Confocal microscopy studies confirmed intracellular localization of targeting systems, resulting in sustained mcDNA uptake. Furthermore, the efficiency of in vitro transfection can be influenced by the presence of R8-mannose, with great implications for gene expression. R8-mannose/PEI/mcDNA ternary systems can be considered valuable tools to instigate further research, aiming for advances in the DNA vaccine field. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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23 pages, 2590 KiB

Open AccessArticle

Hepatocellular-Targeted mRNA Delivery Using Functionalized Selenium Nanoparticles In Vitro

by Dhireshan Singh and Moganavelli Singh

Pharmaceutics 2021, 13(3), 298; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13030298 - 24 Feb 2021

Cited by 29 | Viewed by 3808

Abstract

Selenium’s (Se) chemopreventative and therapeutic properties have attracted attention in nanomedicine. Se nanoparticles (SeNPs) retain these properties of Se while possessing lower toxicity and higher bioavailability, potentiating their use in gene delivery. This study aimed to formulate SeNPs for efficient binding and targeted delivery of FLuc-mRNA to hepatocellular carcinoma cells (HepG2) in vitro. The colorectal adenocarcinoma (Caco-2) and normal human embryonic kidney (HEK293) cells that do not have the asialoorosomucoid receptor (ASGPR) were utilized for comparison. SeNPs were functionalized with chitosan (CS), polyethylene glycol (PEG), and lactobionic acid (LA) for ASGPR targeting on HepG2 cells. Nanoparticles (NPs) and their mRNA-nanocomplexes were characterized by Fourier transform infra-red (FTIR) and UV-vis spectroscopy, transmission electron microscopy (TEM), and nanoparticle tracking analysis (NTA). Gel and fluorescence-based assays assessed the NP’s ability to bind and protect FLuc-mRNA. Cytotoxicity was determined using the -(4,5-dimethythiazol-2-yl)-2,5-diphenyl tetrazolium bromide (MTT) assay, while transgene expression was evaluated using the luciferase reporter gene assay. All NPs appeared spherical with sizes ranging 57.2–130.0 nm and zeta potentials 14.9–31.4 mV. NPs bound, compacted, and protected the mRNA from nuclease digestion and showed negligible cytotoxicity in vitro. Targeted gene expression was highest in the HepG2 cells using the LA targeted NPs. These NPs portend to be efficient nanocarriers of nucleic acids and warrant further investigation. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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Review

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16 pages, 2439 KiB

Open AccessReview

Non-Viral Delivery of RNA Gene Therapy to the Central Nervous System

by Ellen S. Hauck and James G. Hecker

Pharmaceutics 2022, 14(1), 165; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics14010165 - 11 Jan 2022

Cited by 6 | Viewed by 3524

Abstract

Appropriate gene delivery systems are essential for successful gene therapy in clinical medicine. Lipid-mediated nucleic acid delivery is an alternative to viral vector-mediated gene delivery and has the following advantages. Lipid-mediated delivery of DNA or mRNA is usually more rapid than viral-mediated delivery, offers a larger payload, and has a nearly zero risk of incorporation. Lipid-mediated delivery of DNA or RNA is therefore preferable to viral DNA delivery in those clinical applications that do not require long-term expression for chronic conditions. Delivery of RNA may be preferable to non-viral DNA delivery in some clinical applications, since transit across the nuclear membrane is not necessary, and onset of expression with RNA is therefore even faster than with DNA, although both are faster than most viral vectors. Delivery of RNA to target organ(s) has previously been challenging due to RNA’s rapid degradation in biological systems, but cationic lipids complexed with RNA, as well as lipid nanoparticles (LNPs), have allowed for delivery and expression of the complexed RNA both in vitro and in vivo. This review will focus on the non-viral lipid-mediated delivery of RNAs, including mRNA, siRNA, shRNA, and microRNA, to the central nervous system (CNS), an organ with at least two unique challenges. The CNS contains a large number of slowly dividing or non-dividing cell types and is protected by the blood brain barrier (BBB). In non-dividing cells, RNA-lipid complexes demonstrated increased transfection efficiency relative to DNA transfection. The efficiency, timing of the onset, and duration of expression after transfection may determine which nucleic acid is best for which proposed therapy. Expression can be seen as soon as 1 h after RNA delivery, but duration of expression has been limited to 5–7 h. In contrast, transfection with a DNA lipoplex demonstrates protein expression within 5 h and lasts as long as several weeks after transfection. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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13 pages, 1179 KiB

Open AccessReview

Cell-Penetrating Peptides: Emerging Tools for mRNA Delivery

by Hidetomo Yokoo, Makoto Oba and Satoshi Uchida

Pharmaceutics 2022, 14(1), 78; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics14010078 - 29 Dec 2021

Cited by 48 | Viewed by 5883

Abstract

Messenger RNAs (mRNAs) were previously shown to have great potential for preventive vaccination against infectious diseases and therapeutic applications in the treatment of cancers and genetic diseases. Delivery systems for mRNAs, including lipid- and polymer-based carriers, are being developed for improving mRNA bioavailability. Among these systems, cell-penetrating peptides (CPPs) of 4–40 amino acids have emerged as powerful tools for mRNA delivery, which were originally developed to deliver membrane-impermeable drugs, peptides, proteins, and nucleic acids to cells and tissues. Various functionalities can be integrated into CPPs by tuning the composition and sequence of natural and non-natural amino acids for mRNA delivery. With the employment of CPPs, improved endosomal escape efficiencies, selective targeting of dendritic cells (DCs), modulation of endosomal pathways for efficient antigen presentation by DCs, and effective mRNA delivery to the lungs by dry powder inhalation have been reported; additionally, they have been found to prolong protein expression by intracellular stabilization of mRNA. This review highlights the distinctive features of CPP-based mRNA delivery systems. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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26 pages, 2427 KiB

Open AccessReview

Intracellular Routing and Recognition of Lipid-Based mRNA Nanoparticles

by Christophe Delehedde, Luc Even, Patrick Midoux, Chantal Pichon and Federico Perche

Pharmaceutics 2021, 13(7), 945; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13070945 - 24 Jun 2021

Cited by 24 | Viewed by 5478

Abstract

Messenger RNA (mRNA) is being extensively used in gene therapy and vaccination due to its safety over DNA, in the following ways: its lack of integration risk, cytoplasmic expression, and transient expression compatible with fine regulations. However, clinical applications of mRNA are limited by its fast degradation by nucleases, and the activation of detrimental immune responses. Advances in mRNA applications, with the recent approval of COVID-19 vaccines, were fueled by optimization of the mRNA sequence and the development of mRNA delivery systems. Although delivery systems and mRNA sequence optimization have been abundantly reviewed, understanding of the intracellular processing of mRNA is mandatory to improve its applications. We will focus on lipid nanoparticles (LNPs) as they are the most advanced nanocarriers for the delivery of mRNA. Here, we will review how mRNA therapeutic potency can be affected by its interactions with cellular proteins and intracellular distribution. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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14 pages, 776 KiB

Open AccessReview

Protamine-Based Strategies for RNA Transfection

by Natalia Teresa Jarzebska, Mark Mellett, Julia Frei, Thomas M. Kündig and Steve Pascolo

Pharmaceutics 2021, 13(6), 877; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13060877 - 14 Jun 2021

Cited by 39 | Viewed by 6117

Abstract

Protamine is a natural cationic peptide mixture mostly known as a drug for the neutralization of heparin and as a compound in formulations of slow-release insulin. Protamine is also used for cellular delivery of nucleic acids due to opposite charge-driven coupling. This year marks 60 years since the first use of Protamine as a transfection enhancement agent. Since then, Protamine has been broadly used as a stabilization agent for RNA delivery. It has also been involved in several compositions for RNA-based vaccinations in clinical development. Protamine stabilization of RNA shows double functionality: it not only protects RNA from degradation within biological systems, but also enhances penetration into cells. A Protamine-based RNA delivery system is a flexible and versatile platform that can be adjusted according to therapeutic goals: fused with targeting antibodies for precise delivery, digested into a cell penetrating peptide for better transfection efficiency or not-covalently mixed with functional polymers. This manuscript gives an overview of the strategies employed in protamine-based RNA delivery, including the optimization of the nucleic acid’s stability and translational efficiency, as well as the regulation of its immunostimulatory properties from early studies to recent developments. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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10 pages, 627 KiB

Open AccessReview

Minicircles for Investigating and Treating Arthritic Diseases

by Yeri Alice Rim, Yoojun Nam, Narae Park and Ji Hyeon Ju

Pharmaceutics 2021, 13(5), 736; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13050736 - 17 May 2021

Cited by 1 | Viewed by 1993

Abstract

Gene delivery systems have become an essential component of research and the development of therapeutics for various diseases. Minicircles are non-viral vectors with promising characteristics for application in a variety of fields. With their minimal size, minicircles exhibit relatively high safety and efficient delivery of genes of interest into cells. Cartilage tissue lacks the natural ability to heal, making it difficult to treat osteoarthritis (OA) and rheumatoid arthritis (RA), which are the two main types of joint-related disease. Although both OA and RA affect the joint, RA is an autoimmune disease, while OA is a degenerative joint condition. Gene transfer using minicircles has also been used in many studies regarding cartilage and its diseased conditions. In this review, we summarize the cartilage-, OA-, and RA-based studies that have used minicircles as the gene delivery system. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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29 pages, 2390 KiB

Open AccessReview

Multifunctional Immunoadjuvants for Use in Minimalist Nucleic Acid Vaccines

by Saed Abbasi and Satoshi Uchida

Pharmaceutics 2021, 13(5), 644; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13050644 - 01 May 2021

Cited by 16 | Viewed by 4288

Abstract

Subunit vaccines based on antigen-encoding nucleic acids have shown great promise for antigen-specific immunization against cancer and infectious diseases. Vaccines require immunostimulatory adjuvants to activate the innate immune system and trigger specific adaptive immune responses. However, the incorporation of immunoadjuvants into nonviral nucleic acid delivery systems often results in fairly complex structures that are difficult to mass-produce and characterize. In recent years, minimalist approaches have emerged to reduce the number of components used in vaccines. In these approaches, delivery materials, such as lipids and polymers, and/or pDNA/mRNA are designed to simultaneously possess several functionalities of immunostimulatory adjuvants. Such multifunctional immunoadjuvants encode antigens, encapsulate nucleic acids, and control their pharmacokinetic or cellular fate. Herein, we review a diverse class of multifunctional immunoadjuvants in nucleic acid subunit vaccines and provide a detailed description of their mechanisms of adjuvanticity and induction of specific immune responses. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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23 pages, 2291 KiB

Open AccessReview

The Ins and Outs of Messenger RNA Electroporation for Physical Gene Delivery in Immune Cell-Based Therapy

by Diana Campillo-Davo, Maxime De Laere, Gils Roex, Maarten Versteven, Donovan Flumens, Zwi N. Berneman, Viggo F. I. Van Tendeloo, Sébastien Anguille and Eva Lion

Pharmaceutics 2021, 13(3), 396; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13030396 - 16 Mar 2021

Cited by 21 | Viewed by 5396

Abstract

Messenger RNA (mRNA) electroporation is a powerful tool for transient genetic modification of cells. This non-viral method of genetic engineering has been widely used in immunotherapy. Electroporation allows fine-tuning of transfection protocols for each cell type as well as introduction of multiple protein-coding mRNAs at once. As a pioneering group in mRNA electroporation, in this review, we provide an expert overview of the ins and outs of mRNA electroporation, discussing the different parameters involved in mRNA electroporation as well as the production of research-grade and production and application of clinical-grade mRNA for gene transfer in the context of cell-based immunotherapies. Full article

(This article belongs to the Special Issue Delivery Systems of Plasmid DNA and Messenger RNA for Advanced Therapies)

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