2,491 results match your criteria non-viral vectors

Manufacturing NKG2D CAR-T cells with piggyBac transposon vectors and K562 artificial antigen-presenting cells.

Mol Ther Methods Clin Dev 2021 Jun 3;21:107-120. Epub 2021 Mar 3.

Department of Biological Sciences, National University of Singapore, Singapore 117543, Singapore.

Non-viral platforms can be applied rapidly and cost-effectively for chimeric antigen receptor (CAR)-T cell manufacturing. In the present paper, we describe in detail a clinically relevant manufacturing process for NKG2D CAR-T cells through electroporation of CAR-encoding piggyBac transposon plasmids and expansion with K562 artificial antigen-presenting cells. With an optimized protocol, we generated the final cell therapy products with 89. Read More

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Effect of Plasmid DNA Size on Chitosan or Polyethyleneimine Polyplexes Formulation.

Polymers (Basel) 2021 Mar 5;13(5). Epub 2021 Mar 5.

CICS-UBI-Health Sciences Research Centre, Universidade da Beira Interior, Avenida Infante D. Henrique, 6200-506 Covilhã, Portugal.

Gene therapy could be simply defined as a strategy for the introduction of a functional copy of desired genes in patients, to correct some specific mutation and potentially treat the respective disorder. However, this straightforward definition hides very complex processes related to the design and preparation of the therapeutic genes, as well as the development of suitable gene delivery systems. Within non-viral vectors, polymeric nanocarriers have offered an ideal platform to be applied as gene delivery systems. Read More

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Advances in the Formulation and Assembly of Non-Cationic Lipid Nanoparticles for the Medical Application of Gene Therapeutics.

Nanomaterials (Basel) 2021 Mar 23;11(3). Epub 2021 Mar 23.

Department of Surgery, University of Tennessee Graduate School of Medicine, 1924 Alcoa Highway Box U-11, Knoxville, TN 37920, USA.

Lipid nanoparticles have become increasingly popular delivery platforms in the field of gene therapy, but bench-to-bedside success has been limited. Many liposomal gene vectors are comprised of synthetic cationic lipids, which are associated with lipid-induced cytotoxicity and immunogenicity. Natural, non-cationic PEGylated liposomes (PLPs) demonstrate favorable biocompatibility profiles but are not considered viable gene delivery vehicles due to inefficient nucleic acid loading and reduced cellular uptake. Read More

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Recent Advances in Preclinical Research Using PAMAM Dendrimers for Cancer Gene Therapy.

Int J Mol Sci 2021 Mar 13;22(6). Epub 2021 Mar 13.

Department of General Biophysics, Faculty of Biology and Environmental Protection, University of Lodz, Pomorska 141/143, 90-236 Lodz, Poland.

Carriers of genetic material are divided into vectors of viral and non-viral origin. Viral carriers are already successfully used in experimental gene therapies, but despite advantages such as their high transfection efficiency and the wide knowledge of their practical potential, the remaining disadvantages, namely, their low capacity and complex manufacturing process, based on biological systems, are major limitations prior to their broad implementation in the clinical setting. The application of non-viral carriers in gene therapy is one of the available approaches. Read More

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An Overview of Nanocarrier-Based Adjuvants for Vaccine Delivery.

Pharmaceutics 2021 Mar 27;13(4). Epub 2021 Mar 27.

School of Pharmacy and Biomolecular Sciences, Liverpool John Moores University, Liverpool L3 3AF, UK.

The development of vaccines is one of the most significant medical accomplishments which has helped to eradicate a large number of diseases. It has undergone an evolutionary process from live attenuated pathogen vaccine to killed whole organisms or inactivated toxins (toxoids), each of them having its own advantages and disadvantages. The crucial parameters in vaccination are the generation of memory response and protection against infection, while an important aspect is the effective delivery of antigen in an intelligent manner to evoke a robust immune response. Read More

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Non-viral Induction of Transgene-free iPSCs from Somatic Fibroblasts of Multiple Mammalian Species.

Stem Cell Reports 2021 Mar 23. Epub 2021 Mar 23.

Department of Physiology, School of Medicine, Keio University, 35 Shinanomachi, Shinjuku-ku, Tokyo, Japan; Laboratory for Marmoset Neural Architecture, RIKEN Center for Brain Science, Saitama, Japan. Electronic address:

Induced pluripotent stem cells (iPSCs) are capable of providing an unlimited source of cells from all three germ layers and germ cells. The derivation and usage of iPSCs from various animal models may facilitate stem cell-based therapy, gene-modified animal production, and evolutionary studies assessing interspecies differences. However, there is a lack of species-wide methods for deriving iPSCs, in particular by means of non-viral and non-transgene-integrating (NTI) approaches. Read More

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Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies.

Front Pharmacol 2021 16;12:639475. Epub 2021 Mar 16.

School of Women's and Children's Health, Faculty of Medicine, University of New South Wales, Sydney, Australia.

Prognosis of patients with cystic fibrosis (CF) varies extensively despite recent advances in targeted therapies that improve CF transmembrane conductance regulator (CFTR) function. Despite being a multi-organ disease, extensive lung tissue destruction remains the major cause of morbidity and mortality. Progress towards a curative treatment strategy that implements a gene addition-technology to the patients' lungs has been slow and not yet developed beyond clinical trials. Read More

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Current Approaches for Glioma Gene Therapy and Virotherapy.

Front Mol Neurosci 2021 11;14:621831. Epub 2021 Mar 11.

Department of Neurosurgery, University of Michigan Medical School, Ann Arbor, MI, United States.

Glioblastoma (GBM) is the most common and aggressive primary brain tumor in the adult population and it carries a dismal prognosis. Inefficient drug delivery across the blood brain barrier (BBB), an immunosuppressive tumor microenvironment (TME) and development of drug resistance are key barriers to successful glioma treatment. Since gliomas occur through sequential acquisition of genetic alterations, gene therapy, which enables to modification of the genetic make-up of target cells, appears to be a promising approach to overcome the obstacles encountered by current therapeutic strategies. Read More

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Nucleic acids delivered by PEGylated cationic liposomes in systemic lupus erythematosus-prone mice: a possible exacerbation of lupus nephritis in the presence of pre-existing anti-nucleic acid antibodies.

Int J Pharm 2021 Mar 26:120529. Epub 2021 Mar 26.

Department of Pharmacokinetics and Biopharmaceutics, Institute of Biomedical Sciences, Tokushima University, 1-78-1, Sho-machi, Tokushima 770-8505, Japan. Electronic address:

Nucleic acid-based therapy with plasmid DNA (pDNA) and small interfering RNA (siRNA) have received recent attention for their ability to modulate the cellular expression of genes and proteins. Polyethylene glycol-modified (PEGylated) cationic nanoparticles have been used as non-viral vectors for the in vivo delivery of these nucleic acids. We have reported that PEGylated cationic liposomes (PCL) including pDNA or siRNA induce anti-PEG antibodies upon repeated intravenous injection, leading to the formation of immune complexes and enhanced clearance from the blood of subsequent doses. Read More

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Current standards and pitfalls associated with the transfection of primary fibroblast cells.

Biotechnol Prog 2021 Mar 28:e3152. Epub 2021 Mar 28.

Centre for Experimental and Innovative Medicine, Laboratory of Recombinant Proteins Production, University of Agriculture in Krakow, Krakow, Poland.

Cultured fibroblast cells, especially dermal cells, are used for various types of scientific research, particularly within the medical field. Desirable features of the cells include their ease of isolation, rapid cellular growth, and high degree of robustness. Currently, fibroblasts are mainly used to obtain pluripotent cells via a reprogramming process. Read More

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Gene-activated matrix harboring a miR20a-expressing plasmid promotes rat cranial bone augmentation.

Regen Biomater 2021 Mar 13;8(2):rbaa060. Epub 2021 Mar 13.

Department of Regenerative Oral Surgery, Unit of Translational Medicine, Nagasaki University Graduate School of Biomedical Sciences, 1-7-1 Sakamoto, Nagasaki 852-8588, Japan.

Gene-activated matrix (GAM) has a potential usefulness in bone engineering as an alternate strategy for the lasting release of osteogenic proteins but efficient methods to generate non-viral GAM remain to be established. In this study, we investigated whether an atelocollagen-based GAM containing naked-plasmid () DNAs encoding microRNA (miR) 20a, which may promote osteogenesis via multiple pathways associated with the osteogenic differentiation of mesenchymal stem/progenitor cells (MSCs), facilitates rat cranial bone augmentation. First, we confirmed the osteoblastic differentiation functions of generated DNA encoding miR20a (miR20a) , and its transfection regulated the expression of several of target genes, such as Bambi1 and PPARγ, in rat bone marrow MSCs and induced the increased expression of BMP4. Read More

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Therapeutic strategies of recurrent glioblastoma and its molecular pathways 'Lock up the beast'.

Ecancermedicalscience 2021 22;15:1176. Epub 2021 Jan 22.

Alexandria Clinical Oncology Department, Alexandria University, Alexandria 21568, Egypt.

Glioblastoma multiforme (GBM) has a poor prognosis-despite aggressive primary treatment composed of surgery, radiotherapy and chemotherapy, median survival is still around 15 months. It starts to grow again after a year of treatment and eventually nothing is effective at this stage. Recurrent GBM is one of the most disappointing fields for researchers in which their efforts have gained no benefit for patients. Read More

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January 2021

Evaluation of BMP2/miRNA co-expression systems for potent therapeutic efficacy in bone-tissue regeneration.

Eur Cell Mater 2021 Mar 3;41:245-268. Epub 2021 Mar 3.

Donaueschingenstrasse 13, 1200 Vienna,

Reconstruction of bone defects and compensation of deficient repair mechanisms represent important goals within the field of regenerative medicine and require novel safe strategies for translation into the clinic. A non-viral osteogenic gene therapeutic vector system ('hybrid vectors') was generated, combining an improved bone morphogenetic protein 2 (BMP2) gene cassette and single pro-osteogenic microRNAs (miR-148b-3p, miR-20-5p, miR-590b-5p), driven by the U6 promoter. The vectors were tested in vitro for their osteogenic differentiation potential in C2C12 and C3H/10T1/2 cell lines, using BMP2 alone as control. Read More

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The Landscape of Non-Viral Gene Augmentation Strategies for Inherited Retinal Diseases.

Int J Mol Sci 2021 Feb 26;22(5). Epub 2021 Feb 26.

UCL Institute of Ophthalmology, London EC1V 9EL, UK.

Inherited retinal diseases (IRDs) are a heterogeneous group of disorders causing progressive loss of vision, affecting approximately one in 1000 people worldwide. Gene augmentation therapy, which typically involves using adeno-associated viral vectors for delivery of healthy gene copies to affected tissues, has shown great promise as a strategy for the treatment of IRDs. However, the use of viruses is associated with several limitations, including harmful immune responses, genome integration, and limited gene carrying capacity. Read More

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February 2021

Fabrication and Optimization of Linear PEI-Modified Crystal Nanocellulose as an Efficient Non-Viral Vector for Gene Delivery.

Rep Biochem Mol Biol 2020 Oct;9(3):297-308

Nano biotechnology Department, Faculty of Bioscience, Tarbiat Modares University, Tehran, Iran.

Background: One of the major challenges in gene therapy is producing gene carriers that possess high transfection efficiency and low cytotoxicity (1). To achieve this purpose, crystal nanocellulose (CNC) -based nanoparticles grafted with polyethylenimine (PEI) have been developed as an alternative to traditional viral vectors to eliminate potential toxicity and immunogenicity.

Methods: In this study, CNC-PEI10kDa (CNCP) nanoparticles were synthetized and their transfection efficiency was evaluated and compared with linear cationic PEI10kDa (PEI) polymer in HEK293T (HEK) cells. Read More

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October 2020

Emerging role of RNA interference in immune cells engineering and its therapeutic synergism in immunotherapy.

Br J Pharmacol 2021 Apr 17;178(8):1741-1755. Epub 2021 Mar 17.

Institute of Biomedical Engineering and Technology, Shanghai Engineering Research Center of Molecular Therapeutics and New Drug Development, School of Chemistry and Molecular Engineering, East China Normal University, Shanghai, China.

RNAi effectors (e.g. siRNA, shRNA and miRNA) can trigger the silencing of specific genes causing alteration of genomic functions becoming a new therapeutic area for the treatment of infectious diseases, neurodegenerative disorders and cancer. Read More

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Achieving highly efficient gene transfer to the bladder by increasing the molecular weight of polymer-based nanoparticles.

J Control Release 2021 Feb 17;332:210-224. Epub 2021 Feb 17.

School of Pharmacy, University College London, London, UK. Electronic address:

Short dwell-time and poor penetration of the bladder permeability barrier (BPB) are the main obstacles to intravesical treatments for bladder diseases, and is evidenced by the lack of such therapeutic options on the market. Herein, we demonstrate that by finely tuning the molecular weight of our cationic polymer mucoadhesive nanoparticles, we enhanced our gene transfer, leading to improved adherence and penetrance through the BPB in a safe and efficient manner. Specifically, increasing the polymer molecular weight from 45 kDa to 83 kDa enhanced luciferase plasmid transfer to the healthy murine bladder, leading to 1. Read More

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February 2021

Guanidine-rich helical polypeptides bearing hydrophobic amino acid pendants for efficient gene delivery.

Biomater Sci 2021 Apr 19;9(7):2670-2678. Epub 2021 Feb 19.

Institute of Functional Nano & Soft Materials (FUNSOM), Collaborative Innovation Center of Suzhou Nano Science and Technology, Soochow University, Suzhou 215123, China.

Non-viral gene delivery vectors with high transfection efficiency both in vitro and in vivo and low cytotoxicity are highly desirable for clinical applications. Herein, a series of guanidine-rich polypeptides bearing hydrophobic amino acid pendants was efficiently prepared via the 1,3-dipolar cycloaddition between azido decorated polypeptide and propargyl functionalized guanidinium and N-acetylamino acids. CD analysis indicated α-helical conformations of all resulting polypeptides in aqueous solution. Read More

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Targeted Dual Small Interfering Ribonucleic Acid Delivery via Non-Viral Polymeric Vectors for Pulmonary Fibrosis Therapy.

Adv Mater 2021 Mar 19;33(12):e2007798. Epub 2021 Feb 19.

Yong Loo Lin School of Medicine and Faculty of Engineering, National University of Singapore, Singapore, 119228, Singapore.

Inhibiting the myofibroblast differentiation of lung-resident mesenchymal stem cells (LR-MSCs) is a promising yet challenging approach for pulmonary fibrosis (PF) therapy. Here, micelles formed by a graft copolymer of multiple PEGs modified branched polyethylenimine are used for delivering runt-related transcription factor-1 (RUNX1) small interfering RNA (siRNA) (siRUNX1) to the lung, aiming to inhibit the myofibroblast differentiation of LR-MSCs. LR-MSC targeting is achieved by functionalizing the micelle surface with an anti-stem-cell antigen-1 antibody fragment (Fab'). Read More

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Inner Ear Gene Delivery: Vectors and Routes.

Hearing Balance Commun 2020 25;18(4):278-285. Epub 2020 Aug 25.

Department of Otolaryngology -- Head and Neck Surgery, Columbia University Vagelos College of Physicians and Surgeons, New York, NY.

Objectives: Current treatments for hearing loss offer some functional improvements in hearing, but do not restore normal hearing. The aim of this review is to highlight recent advances in viral and non-viral vectors for gene therapy and to discuss approaches for overcoming barriers inherent to inner ear delivery of gene products.

Data Sources: The databases used were Medline, EMBASE, Web of Science, and Google Scholar. Read More

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Layer-by-Layer technique as a versatile tool for gene delivery applications.

Expert Opin Drug Deliv 2021 Feb 18:1-19. Epub 2021 Feb 18.

Laboratory of Micro-Encapsulation and Targeted Delivery of Biologically Active Compounds, Peter The Great St. Petersburg Polytechnic University , St. Petersburg, Russia.

: Gene therapy is a breakthrough medical field which focuses on the therapeutic delivery of recombinant nucleic acids in order to treat or prevent a broad spectrum of diseases. However, a number of important obstacles remain before its wide introduction into clinical practice can be envisaged. One of the biggest bottlenecks is the lack of efficient and safe delivery technologies, particularly, for distribution. Read More

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February 2021

The combined disulfide cross-linking and tyrosine-modification of very low molecular weight linear PEI synergistically enhances transfection efficacies and improves biocompatibility.

Eur J Pharm Biopharm 2021 Apr 11;161:56-65. Epub 2021 Feb 11.

Rudolf-Boehm-Institute for Pharmacology and Toxicology, Clinical Pharmacology, Leipzig University, Faculty of Medicine, Leipzig, Germany. Electronic address:

Efficient and non-toxic DNA delivery is still a major limiting factor for non-viral gene therapy. Among the large diversity of non-viral vectors, the cationic polymer polyethylenimine (PEI) plays a prominent role in nucleic acid delivery. Since higher molecular weight of PEI is beneficial for transfection efficacy, but also leads to higher cytotoxicity, the biodegradable cross-linking of low-molecular PEIs, e. Read More

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Improved DNA delivery using invasive E. coli DH10B in human cells by modified bactofection method.

J Control Release 2021 Feb 6;332:233-244. Epub 2021 Feb 6.

Griffith Institute for Drug Discovery, Griffith University, Queensland, Australia. Electronic address:

E. coli mediated gene delivery faces a major drawback of low efficiency despite of being a safer alternative to viral vectors. This study showed a novel, simple and effective strategy to enhance invasive E. Read More

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February 2021

Gene therapy for inherited metabolic diseases.

J Mother Child 2020 Nov 10;24(2):53-64. Epub 2020 Nov 10.

Genetics and Genomic Medicine, Great Ormond Street Institute of Child Health, University College London, London, UK.

Over the last two decades, gene therapy has been successfully translated to many rare diseases. The number of clinical trials is rapidly expanding and some gene therapy products have now received market authorisation in the western world. Inherited metabolic diseases (IMD) are orphan diseases frequently associated with a severe debilitating phenotype with limited therapeutic perspective. Read More

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November 2020

Lipid Nanoparticles as Delivery Systems for RNA-Based Vaccines.

Pharmaceutics 2021 Feb 2;13(2). Epub 2021 Feb 2.

Department of Pharmaceutics, College of Pharmacy, King Saud University, Riyadh 11495, Saudi Arabia.

There has been increased interest in the development of RNA-based vaccines for protection against various infectious diseases and also for cancer immunotherapies. Rapid and cost-effective manufacturing methods in addition to potent immune responses observed in preclinical and clinical studies have made mRNA-based vaccines promising alternatives to conventional vaccine technologies. However, efficient delivery of these vaccines requires that the mRNA be protected against extracellular degradation. Read More

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February 2021

Strategies for cancer gene-delivery improvement by non-viral vectors.

Int J Pharm 2021 Mar 29;596:120291. Epub 2021 Jan 29.

Department of Pharmaceutical Technology and Chemistry, School of Pharmacy and Nutrition, University of Navarra, 31080 Pamplona, Spain. Electronic address:

Lack of selectivity together with severe side effects in conventional cancer treatment have afforded the development of new strategies based on gene therapy. Nowadays, gene therapy is employed through both viral and non-viral vectors. In spite of the high transfection activity of viral vectors, some drawbacks have pointed out to non-viral vectors as a safer alternative. Read More

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The future of retinal gene therapy: evolving from subretinal to intravitreal vector delivery.

Maya Ross Ron Ofri

Neural Regen Res 2021 Sep;16(9):1751-1759

Koret School of Veterinary Medicine, The Hebrew University of Jerusalem, Rehovot, Israel.

Inherited retinal degenerations are a leading and untreatbale cause of blindness, and as such they are targets for gene therapy. Numerous gene therapy treatments have progressed from laboratory research to clinical trails, and a pioneering gene therapy received the first ever FDA approval for treating patients. However, currently retinal gene therapy mostly involves subretinal injection of the therapeutic agent, which treats a limited area, entails retinal detachment and other potential complications, and requires general anesthesia with consequent risks, costs and prolonged recovery. Read More

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September 2021

Targeted delivery of therapeutic agents to the heart.

Nat Rev Cardiol 2021 Jan 26. Epub 2021 Jan 26.

Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

For therapeutic materials to be successfully delivered to the heart, several barriers need to be overcome, including the anatomical challenges of access, the mechanical force of the blood flow, the endothelial barrier, the cellular barrier and the immune response. Various vectors and delivery methods have been proposed to improve the cardiac-specific uptake of materials to modify gene expression. Viral and non-viral vectors are widely used to deliver genetic materials, but each has its respective advantages and shortcomings. Read More

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January 2021

Strategies for Vaccination: Conventional Vaccine Approaches Versus New-Generation Strategies in Combination with Adjuvants.

Pharmaceutics 2021 Jan 22;13(2). Epub 2021 Jan 22.

Biology Department, Aljumum University College, Umm Al-Qura University, Makkah 21955, Saudi Arabia.

The current COVID-19 pandemic, caused by severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2), has raised significant economic, social, and psychological concerns. The rapid spread of the virus, coupled with the absence of vaccines and antiviral treatments for SARS-CoV-2, has galvanized a major global endeavor to develop effective vaccines. Within a matter of just a few months of the initial outbreak, research teams worldwide, adopting a range of different strategies, embarked on a quest to develop effective vaccine that could be effectively used to suppress this virulent pathogen. Read More

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January 2021

Stabilization of Poly (β-Amino Ester) Nanoparticles for the Efficient Intracellular Delivery of PiggyBac Transposon.

Bioengineering (Basel) 2021 Jan 20;8(2). Epub 2021 Jan 20.

Department of Biomedical Engineering and Chemical Engineering, University of Texas at San Antonio, One UTSA Circle, San Antonio, TX 78249, USA.

The administration of gene-editing tools has been proposed as a promising therapeutic approach for correcting mutations that cause diseases. Gene-editing tools, composed of relatively large plasmid DNA constructs that often need to be co-delivered with a guiding protein, are unable to spontaneously penetrate mammalian cells. Although viral vectors facilitate DNA delivery, they are restricted by the size of the plasmid to carry. Read More

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January 2021