Non-Viral Gene Delivery Systems

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

Deadline for manuscript submissions: closed (30 June 2020) | Viewed by 70141

Special Issue Editor

Center for Neuroscience and Cell Biology, University of Coimbra, 3004-517 Coimbra, Portugal
Interests: nanosystems; gene delivery; drug delivery; cancer gene therapy; cancer therapy
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Special Issue Information

Dear Colleagues,

Advances in the field of gene therapy have significantly improved the possibility for nucleic acids as highly promising agents for the treatment of both inherited and acquired human diseases. Substantial progress has been made in the development of different types of nucleic acids, including plasmid DNA, mRNA, microRNA, small interfering RNA, and antisense oligonucleotides. Nevertheless, despite the immense pharmacological potential of these molecules, the successful clinical application of genetic material-based strategies remains dependent on the generation of safe and effective delivery systems that have the ability to overcome the numerous biological barriers associated with gene delivery into target cells.

Until now, the large majority of gene therapy clinical trials have been based on the use of viral vectors, namely, due to features such as high levels of transduction, or efficient and stable integration of exogenous DNA into host genomes. However, several drawbacks have been associated with viral vectors, such as immunogenicity, carcinogenesis, the size limit of exogenous DNA, and the difficulty of large-scale production. Non-viral gene delivery systems have the potential to overcome these limitations, allowing not only a safe but also an efficient gene delivery process into target cells.

This Special Issue has the aim of highlighting the current progress in non-viral gene delivery systems. In this regard, I would like to invite you to submit your original papers or reviews on the design, development, characterization, and application of non-viral gene delivery systems.

Dr. Henrique Faneca
Guest Editor

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Keywords

  • Non-viral vectors
  • Lipid-based systems
  • Polymer-based systems
  • Gene delivery
  • Transfection

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Published Papers (13 papers)

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Editorial

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4 pages, 179 KiB  
Editorial
Non-Viral Gene Delivery Systems
by Henrique Faneca
Pharmaceutics 2021, 13(4), 446; https://doi.org/10.3390/pharmaceutics13040446 - 26 Mar 2021
Cited by 9 | Viewed by 1881
Abstract
The advances in the field of gene therapy have significantly improved the possibility for nucleic acids as highly promising agents for the treatment of both inherited and acquired human diseases [...] Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)

Research

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25 pages, 8603 KiB  
Article
Development of iRGD-Modified Peptide Carriers for Suicide Gene Therapy of Uterine Leiomyoma
by Anna Egorova, Sofia Shtykalova, Alexander Selutin, Natalia Shved, Marianna Maretina, Sergei Selkov, Vladislav Baranov and Anton Kiselev
Pharmaceutics 2021, 13(2), 202; https://doi.org/10.3390/pharmaceutics13020202 - 02 Feb 2021
Cited by 10 | Viewed by 2026
Abstract
Uterine leiomyoma (UL) is one of the most common benign tumors in women that often leads to many reproductive complications. Suicide genetherapy was suggested as a promising approach for UL treatment. In the present study, we describe iRGD ligand-conjugated cysteine-rich peptide carrier RGD1-R6 [...] Read more.
Uterine leiomyoma (UL) is one of the most common benign tumors in women that often leads to many reproductive complications. Suicide genetherapy was suggested as a promising approach for UL treatment. In the present study, we describe iRGD ligand-conjugated cysteine-rich peptide carrier RGD1-R6 for targeted DNA delivery to αvβ3 integrin-expressing primary UL cells. The physico-chemical properties, cytotoxicity, transfection efficiency and specificity of DNA/RGD1-R6 polyplexes were investigated. TheHSV-1thymidine kinase encoding plasmid delivery to PANC-1pancreatic carcinoma cells and primary UL cells resulted in significant suicide gene therapy effects. Subsequent ganciclovir treatment decreased cells proliferative activity, induced of apoptosis and promoted cells death.The obtained results allow us to concludethatthe developed RGD1-R6 carrier can be considered a promising candidate for suicide gene therapy of uterine leiomyoma. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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16 pages, 2240 KiB  
Article
Sphingomyelin-Based Nanosystems (SNs) for the Development of Anticancer miRNA Therapeutics
by Surasa Nagachinta, Belen Lopez Bouzo, Abi Judit Vazquez-Rios, Rafael Lopez and Maria de la Fuente
Pharmaceutics 2020, 12(2), 189; https://doi.org/10.3390/pharmaceutics12020189 - 22 Feb 2020
Cited by 31 | Viewed by 3646
Abstract
Gene replacement therapy with oncosuppressor microRNAs (miRNAs) is a promising alternative to interfere with cancer progression. However, miRNAs are highly inefficient in a biological environment, hampering a successful translation to clinics. Nanotechnology can tackle this drawback by providing delivery systems able to efficiently [...] Read more.
Gene replacement therapy with oncosuppressor microRNAs (miRNAs) is a promising alternative to interfere with cancer progression. However, miRNAs are highly inefficient in a biological environment, hampering a successful translation to clinics. Nanotechnology can tackle this drawback by providing delivery systems able to efficiently deliver them to cancer cells. Thus, the objective of this work was to develop biocompatible nanosystems based on sphingomyelin (SM) for the intracellular delivery of miRNAs to colorectal cancer cells. We pursued two different approaches to select the most appropriate composition for miRNA delivery. On the one hand, we prepared sphingomyelin-based nanosystems (SNs) that incorporate the cationic lipid stearylamine (ST) to support the association of miRNA by the establishment of electrostatic interactions (SNs–ST). On the other hand, the cationic surfactant (DOTAP) was used to preform lipidic complexes with miRNA (Lpx), which were further encapsulated into SNs (SNs-Lpx). Restitution of miRNA145 levels after transfection with SNs-Lpx was related to the strongest anticancer effect in terms of tumor proliferation, colony forming, and migration capacity assays. Altogether, our results suggest that SNs have the potential for miRNA delivery to develop innovative anticancer therapies. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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14 pages, 5508 KiB  
Article
Anti-Metastatic Effects on Melanoma via Intravenous Administration of Anti-NF-κB siRNA Complexed with Functional Peptide-Modified Nano-Micelles
by Hisako Ibaraki, Takanori Kanazawa, Minami Owada, Keiko Iwaya, Yuuki Takashima and Yasuo Seta
Pharmaceutics 2020, 12(1), 64; https://doi.org/10.3390/pharmaceutics12010064 - 15 Jan 2020
Cited by 23 | Viewed by 3676
Abstract
Controlling metastasis is an important strategy in cancer treatment. Nanotechnology and nucleic acids with novel modalities are promising regulators of cancer metastasis. We aimed to develop a small interfering RNA (siRNA) systemic delivery and anti-metastasis system using nanotechnology. We previously reported that polyethylene [...] Read more.
Controlling metastasis is an important strategy in cancer treatment. Nanotechnology and nucleic acids with novel modalities are promising regulators of cancer metastasis. We aimed to develop a small interfering RNA (siRNA) systemic delivery and anti-metastasis system using nanotechnology. We previously reported that polyethylene glycol-polycaprolactone (PEG-PCL) and functional peptide CH2R4H2C nano-micelle (MPEG-PCL-CH2R4H2C) has high siRNA silencing effects, indicated by increased drug accumulation in tumor-bearing mice, and has an anti-tumor effect on solid tumors upon systemic injection. In this study, we aimed to apply our micelles to inhibit metastasis and evaluated the inhibitory effect of anti-RelA siRNA (siRelA), which is a subunit of NF-κB conjugated with MPEG-PCL-CH2R4H2C, via systemic administration. We report that siRelA/MPEG-PCL-CH2R4H2C had a high cellular uptake and suppressed the migration/invasion of cells in B16F10 cells without toxicity. In addition, in a lung metastasis mouse model using intravenous administration of B16F10 cells treated with siRelA/MPEG-PCL-CH2R4H2C, the number of lung nodules in lung tissue significantly decreased compared to naked siRelA and siControl/MPEG-PCL-CH2R4H2C micelle treatments. Hence, we show that RelA expression can reduce cancer metastasis, and MPEG-PCL-CH2R4H2C is an effective siRNA carrier for anti-metastasis cancer therapies. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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19 pages, 5172 KiB  
Article
Sugar and Polymer Excipients Enhance Uptake and Splice-Switching Activity of Peptide-Dendrimer/Lipid/Oligonucleotide Formulations
by Osama Saher, Taavi Lehto, Olof Gissberg, Dhanu Gupta, Oskar Gustafsson, Samir EL Andaloussi, Tamis Darbre, Karin E. Lundin, C. I. Edvard Smith and Rula Zain
Pharmaceutics 2019, 11(12), 666; https://doi.org/10.3390/pharmaceutics11120666 - 09 Dec 2019
Cited by 8 | Viewed by 3816
Abstract
Non-viral transfection vectors are commonly used for oligonucleotide (ON) delivery but face many challenges before reaching the desired compartments inside cells. With the support of additional compounds, it might be more feasible for a vector to endure the barriers and achieve efficient delivery. [...] Read more.
Non-viral transfection vectors are commonly used for oligonucleotide (ON) delivery but face many challenges before reaching the desired compartments inside cells. With the support of additional compounds, it might be more feasible for a vector to endure the barriers and achieve efficient delivery. In this report, we screened 18 different excipients and evaluated their effect on the performance of peptide dendrimer/lipid vector to deliver single-stranded, splice-switching ONs under serum conditions. Transfection efficiency was monitored in four different reporter cell lines by measuring splice-switching activity on RNA and protein levels. All reporter cell lines used had a mutated human β-globin intron 2 sequence interrupting the luciferase gene, which led to an aberrant splicing of luciferase pre-mRNA and subsidence of luciferase protein translation. In the HeLa Luc/705 reporter cell line (a cervical cancer cell line), the lead excipients (Polyvinyl derivatives) potentiated the splice-switching activity up to 95-fold, compared to untreated cells with no detected cytotoxicity. Physical characterization revealed that lead excipients decreased the particle size and the zeta potential of the formulations. In vivo biodistribution studies emphasized the influence of formulations as well as the type of excipients on biodistribution profiles of the ON. Subsequently, we suggest that the highlighted impact of tested excipients would potentially assist in formulation development to deliver ON therapeutics in pre-clinical and clinical settings. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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16 pages, 3195 KiB  
Article
A Non-Viral Plasmid DNA Delivery System Consisting on a Lysine-Derived Cationic Lipid Mixed with a Fusogenic Lipid
by María Martínez-Negro, Natalia Sánchez-Arribas, Andrés Guerrero-Martínez, María Luisa Moyá, Conchita Tros de Ilarduya, Francisco Mendicuti, Emilio Aicart and Elena Junquera
Pharmaceutics 2019, 11(12), 632; https://doi.org/10.3390/pharmaceutics11120632 - 27 Nov 2019
Cited by 13 | Viewed by 3203
Abstract
The insertion of biocompatible amino acid moieties in non-viral gene nanocarriers is an attractive approach that has been recently gaining interest. In this work, a cationic lipid, consisting of a lysine-derived moiety linked to a C12 chain (LYCl) was combined with a [...] Read more.
The insertion of biocompatible amino acid moieties in non-viral gene nanocarriers is an attractive approach that has been recently gaining interest. In this work, a cationic lipid, consisting of a lysine-derived moiety linked to a C12 chain (LYCl) was combined with a common fusogenic helper lipid (DOPE) and evaluated as a potential vehicle to transfect two plasmid DNAs (encoding green fluorescent protein GFP and luciferase) into COS-7 cells. A multidisciplinary approach has been followed: (i) biophysical characterization based on zeta potential, gel electrophoresis, small-angle X-ray scattering (SAXS), and cryo-transmission electronic microscopy (cryo-TEM); (ii) biological studies by fluorescence assisted cell sorting (FACS), luminometry, and cytotoxicity experiments; and (iii) a computational study of the formation of lipid bilayers and their subsequent stabilization with DNA. The results indicate that LYCl/DOPE nanocarriers are capable of compacting the pDNAs and protecting them efficiently against DNase I degradation, by forming Lα lyotropic liquid crystal phases, with an average size of ~200 nm and low polydispersity that facilitate the cellular uptake process. The computational results confirmed that the LYCl/DOPE lipid bilayers are stable and also capable of stabilizing DNA fragments via lipoplex formation, with dimensions consistent with experimental values. The optimum formulations (found at 20% of LYCl content) were able to complete the transfection process efficiently and with high cell viabilities, even improving the outcomes of the positive control Lipo2000*. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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18 pages, 3430 KiB  
Article
An Effective Cationic Human Serum Albumin-Based Gene-Delivery Carrier Containing the Nuclear Localization Signal
by Guannan Guan, Baohui Song, Jie Zhang, Kang Chen, Haiyang Hu, Mingyue Wang and Dawei Chen
Pharmaceutics 2019, 11(11), 608; https://doi.org/10.3390/pharmaceutics11110608 - 13 Nov 2019
Cited by 15 | Viewed by 3278
Abstract
Considerable effort has been devoted to the development of gene carriers over the years. However, toxicity, immunogenicity, and low transfection efficiency are still major barriers. How to overcome these obstacles has become a burning question in gene delivery. In the present study, a [...] Read more.
Considerable effort has been devoted to the development of gene carriers over the years. However, toxicity, immunogenicity, and low transfection efficiency are still major barriers. How to overcome these obstacles has become a burning question in gene delivery. In the present study, a simple cationic human serum albumin (CHSA)-based gene-delivery system containing nuclear localization signals (NLSs) was constructed to conquer the limitations. CHSA/NLS/plasmid DNA (pDNA) complexes were prepared and characterized by Hoechst 33258 intercalation, gel retardation assay, morphological analysis, circular dichroism (CD) spectroscopy, particle size, and zeta potential measurements. Results showed that CHSA/NLS/pDNA complexes were able to condense and protect pDNA with high encapsulation efficiency. The complexes displayed a nutritional effect on cells at a low concentration and there was no significant cytotoxicity or immunogenicity. In addition, CHSA/NLS/pDNA complexes exhibited excellent cellular uptake rates and the mechanism was mainly the clathrin or macropinocytosis-dependent endocytosis pathway. Furthermore, CHSA/NLS/pDNA significantly enhanced gene expression efficiency in vitro. More importantly, CHSA/NLS/pDNA complexes showed a desired antitumor effect in vivo, exhibiting the highest inhibition rate (57.3%) and significant upregulation in p53 protein. All these results confirm that CHSA/NLS/pDNA complexes have a bright future as a safe and effective delivery system for gene therapy. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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Review

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20 pages, 3511 KiB  
Review
Synthetic Virus-Derived Nanosystems (SVNs) for Delivery and Precision Docking of Large Multifunctional DNA Circuitry in Mammalian Cells
by Francesco Aulicino, Julien Capin and Imre Berger
Pharmaceutics 2020, 12(8), 759; https://doi.org/10.3390/pharmaceutics12080759 - 11 Aug 2020
Cited by 11 | Viewed by 5543
Abstract
DNA delivery is at the forefront of current research efforts in gene therapy and synthetic biology. Viral vectors have traditionally dominated the field; however, nonviral delivery systems are increasingly gaining traction. Baculoviruses are arthropod-specific viruses that can be easily engineered and repurposed to [...] Read more.
DNA delivery is at the forefront of current research efforts in gene therapy and synthetic biology. Viral vectors have traditionally dominated the field; however, nonviral delivery systems are increasingly gaining traction. Baculoviruses are arthropod-specific viruses that can be easily engineered and repurposed to accommodate and deliver large sequences of exogenous DNA into mammalian cells, tissues, or ultimately organisms. These synthetic virus-derived nanosystems (SVNs) are safe, readily customized, and can be manufactured at scale. By implementing clustered regularly interspaced palindromic repeats (CRISPR) associated protein (CRISPR/Cas) modalities into this system, we developed SVNs capable of inserting complex DNAs into genomes, at base pair precision. We anticipate a major role for SVNs as an attractive alternative to viral vectors in accelerating genome engineering and gene therapy applications in the future. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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21 pages, 1865 KiB  
Review
Hydrogel-Based Localized Nonviral Gene Delivery in Regenerative Medicine Approaches—An Overview
by Natalia Carballo-Pedrares, Isaac Fuentes-Boquete, Silvia Díaz-Prado and Ana Rey-Rico
Pharmaceutics 2020, 12(8), 752; https://doi.org/10.3390/pharmaceutics12080752 - 10 Aug 2020
Cited by 30 | Viewed by 3866
Abstract
Hydrogel-based nonviral gene delivery constitutes a powerful strategy in various regenerative medicine scenarios, as those concerning the treatment of musculoskeletal, cardiovascular, or neural tissues disorders as well as wound healing. By a minimally invasive administration, these systems can provide a spatially and temporarily [...] Read more.
Hydrogel-based nonviral gene delivery constitutes a powerful strategy in various regenerative medicine scenarios, as those concerning the treatment of musculoskeletal, cardiovascular, or neural tissues disorders as well as wound healing. By a minimally invasive administration, these systems can provide a spatially and temporarily defined supply of specific gene sequences into the target tissue cells that are overexpressing or silencing the original gene, which can promote natural repairing mechanisms to achieve the desired effect. In the present work, we provide an overview of the most avant-garde approaches using various hydrogels systems for controlled delivery of therapeutic nucleic acid molecules in different regenerative medicine approaches. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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45 pages, 9449 KiB  
Review
Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications
by Ana Maria Carvalho, Rosemeyre A. Cordeiro and Henrique Faneca
Pharmaceutics 2020, 12(7), 649; https://doi.org/10.3390/pharmaceutics12070649 - 09 Jul 2020
Cited by 27 | Viewed by 4551
Abstract
Advances in gene therapy have been foreshadowing its potential for the treatment of a vast range of diseases involving genetic malfunctioning. However, its therapeutic efficiency and successful outcome are highly dependent on the development of the ideal gene delivery system. On that matter, [...] Read more.
Advances in gene therapy have been foreshadowing its potential for the treatment of a vast range of diseases involving genetic malfunctioning. However, its therapeutic efficiency and successful outcome are highly dependent on the development of the ideal gene delivery system. On that matter, silica-based vectors have diverted some attention from viral and other types of non-viral vectors due to their increased safety, easily modifiable structure and surface, high stability, and cost-effectiveness. The versatility of silane chemistry and the combination of silica with other materials, such as polymers, lipids, or inorganic particles, has resulted in the development of carriers with great loading capacities, ability to effectively protect and bind genetic material, targeted delivery, and stimuli-responsive release of cargos. Promising results have been obtained both in vitro and in vivo using these nanosystems as multifunctional platforms in different potential therapeutic areas, such as cancer or brain therapies, sometimes combined with imaging functions. Herein, the current advances in silica-based systems designed for gene therapy are reviewed, including their main properties, fabrication methods, surface modifications, and potential therapeutic applications. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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20 pages, 1523 KiB  
Review
piggyBac-Based Non-Viral In Vivo Gene Delivery Useful for Production of Genetically Modified Animals and Organs
by Masahiro Sato, Emi Inada, Issei Saitoh, Satoshi Watanabe and Shingo Nakamura
Pharmaceutics 2020, 12(3), 277; https://doi.org/10.3390/pharmaceutics12030277 - 19 Mar 2020
Cited by 16 | Viewed by 5832
Abstract
In vivo gene delivery involves direct injection of nucleic acids (NAs) into tissues, organs, or tail-veins. It has been recognized as a useful tool for evaluating the function of a gene of interest (GOI), creating models for human disease and basic research targeting [...] Read more.
In vivo gene delivery involves direct injection of nucleic acids (NAs) into tissues, organs, or tail-veins. It has been recognized as a useful tool for evaluating the function of a gene of interest (GOI), creating models for human disease and basic research targeting gene therapy. Cargo frequently used for gene delivery are largely divided into viral and non-viral vectors. Viral vectors have strong infectious activity and do not require the use of instruments or reagents helpful for gene delivery but bear immunological and tumorigenic problems. In contrast, non-viral vectors strictly require instruments (i.e., electroporator) or reagents (i.e., liposomes) for enhanced uptake of NAs by cells and are often accompanied by weak transfection activity, with less immunological and tumorigenic problems. Chromosomal integration of GOI-bearing transgenes would be ideal for achieving long-term expression of GOI. piggyBac (PB), one of three transposons (PB, Sleeping Beauty (SB), and Tol2) found thus far, has been used for efficient transfection of GOI in various mammalian cells in vitro and in vivo. In this review, we outline recent achievements of PB-based production of genetically modified animals and organs and will provide some experimental concepts using this system. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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34 pages, 2162 KiB  
Review
Gene Therapy in Cancer Treatment: Why Go Nano?
by Catarina Roma-Rodrigues, Lorenzo Rivas-García, Pedro V. Baptista and Alexandra R. Fernandes
Pharmaceutics 2020, 12(3), 233; https://doi.org/10.3390/pharmaceutics12030233 - 05 Mar 2020
Cited by 120 | Viewed by 17813
Abstract
The proposal of gene therapy to tackle cancer development has been instrumental for the development of novel approaches and strategies to fight this disease, but the efficacy of the proposed strategies has still fallen short of delivering the full potential of gene therapy [...] Read more.
The proposal of gene therapy to tackle cancer development has been instrumental for the development of novel approaches and strategies to fight this disease, but the efficacy of the proposed strategies has still fallen short of delivering the full potential of gene therapy in the clinic. Despite the plethora of gene modulation approaches, e.g., gene silencing, antisense therapy, RNA interference, gene and genome editing, finding a way to efficiently deliver these effectors to the desired cell and tissue has been a challenge. Nanomedicine has put forward several innovative platforms to overcome this obstacle. Most of these platforms rely on the application of nanoscale structures, with particular focus on nanoparticles. Herein, we review the current trends on the use of nanoparticles designed for cancer gene therapy, including inorganic, organic, or biological (e.g., exosomes) variants, in clinical development and their progress towards clinical applications. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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21 pages, 2284 KiB  
Review
Cell Penetrating Peptides, Novel Vectors for Gene Therapy
by Rebecca E. Taylor and Maliha Zahid
Pharmaceutics 2020, 12(3), 225; https://doi.org/10.3390/pharmaceutics12030225 - 03 Mar 2020
Cited by 107 | Viewed by 9702
Abstract
Cell penetrating peptides (CPPs), also known as protein transduction domains (PTDs), first identified ~25 years ago, are small, 6–30 amino acid long, synthetic, or naturally occurring peptides, able to carry variety of cargoes across the cellular membranes in an intact, functional form. Since [...] Read more.
Cell penetrating peptides (CPPs), also known as protein transduction domains (PTDs), first identified ~25 years ago, are small, 6–30 amino acid long, synthetic, or naturally occurring peptides, able to carry variety of cargoes across the cellular membranes in an intact, functional form. Since their initial description and characterization, the field of cell penetrating peptides as vectors has exploded. The cargoes they can deliver range from other small peptides, full-length proteins, nucleic acids including RNA and DNA, liposomes, nanoparticles, and viral particles as well as radioisotopes and other fluorescent probes for imaging purposes. In this review, we will focus briefly on their history, classification system, and mechanism of transduction followed by a summary of the existing literature on use of CPPs as gene delivery vectors either in the form of modified viruses, plasmid DNA, small interfering RNA, oligonucleotides, full-length genes, DNA origami or peptide nucleic acids. Full article
(This article belongs to the Special Issue Non-Viral Gene Delivery Systems)
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