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J Inflamm Res 2021 1;14:1257-1270. Epub 2021 Apr 1.

Blacktown Clinical School, Western Sydney University, Blacktown, NSW, 2148, Australia.

Introduction: Interferon lambdas (IFN-λs) are antiviral cytokines that restrict pathogen infection and dissemination at barrier surfaces. Controlled expression of IFN-λs efficiently eliminates acute infections by activating a suite of interferon stimulated genes that inhibit viral propagation and activate local immune cells. Excessive or prolonged production of IFN-λs can however mediate tissue inflammation and disrupt epithelial barriers in both viral and non-viral disease. Read More

Mol Ther Oncolytics 2021 Mar 5;20:646-658. Epub 2021 Mar 5.

Department of Pediatrics, Graduate School of Medicine, Kyoto Prefectural University of Medicine, Kyoto 602-8566, Japan.

Ephrin type-B receptor 4 (EPHB4), expressed in tumors including rhabdomyosarcoma, is a suitable target for chimeric antigen receptor (CAR)-T cells. Ligand-independent activation of EPHB4 causes cell proliferation and malignant transformation in rhabdomyosarcoma, whereas ligand-dependent stimulation of EPHB4 induces apoptosis in rhabdomyosarcoma. Therefore, we hypothesized that ligand-based, EPHB4-specific CAR-T cells may kill rhabdomyosarcoma cells without stimulating downstream cell proliferation mechanisms. Read More

Acta Biomater 2021 Apr 1. Epub 2021 Apr 1.

Department of Biomedical Sciences, Texas A&M University College of Dentistry, Dallas, TX 75246, USA. Electronic address:

Dental pulp is a highly vascularized tissue, situated in an inextensible environment surrounded by rigid dentinal walls. The pulp receives its blood supply solely from the small apical foramen of a tooth root. Due to the unique anatomy that controls nutrition supply, regeneration of pulp tissue in a full-length tooth root has long been a challenge in regenerative endodontics. Read More

Int J Pharm 2021 Apr 1:120566. Epub 2021 Apr 1.

Centre de Biophysique Moléculaire, CNRS UPR4301, Inserm and University of Orléans, F-45071, Orléans cedex 02, France. Electronic address:

Here, we report the synthesis of 3,6,9-trioxaundecan-1-{4-[(2-Chloroethyl)Ethylamino)]-Benzylamino},11-Azide (CEBA). CEBA alkylates the N7 of guanine of DNA thanks its chloroethyl group and can be coupled by a strain-promoted azide-alkyne cycloaddition to an alkynylated molecule. The optimization of the alkylation level of pDNA reveals that the expression of the encoded gene is preserved when it is randomly modified with at most 1 CEBA molecule per 150 bp. Read More

Pharmaceutics 2021 Mar 26;13(4). Epub 2021 Mar 26.

CNC-Center for Neuroscience and Cell Biology, University of Coimbra, 3004-517 Coimbra, Portugal.

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 [... Read More

Pharmaceutics 2021 Mar 22;13(3). Epub 2021 Mar 22.

Grupo de Diseño de Productos y Procesos (GDPP), Department of Chemical and Food Engineering, Universidad de los Andes, Bogotá 111711, Colombia.

Gene therapy has been used as a potential approach to address the diagnosis and treatment of genetic diseases and inherited disorders. In this line, non-viral systems have been exploited as promising alternatives for delivering therapeutic transgenes and proteins. In this review, we explored how biological barriers are effectively overcome by non-viral systems, usually nanoparticles, to reach an efficient delivery of cargoes. Read More

Pharmaceutics 2021 Mar 16;13(3). Epub 2021 Mar 16.

Tumor Immunology Group, Laboratory of Experimental Hematology, Faculty of Medicine and Health Sciences, Vaccine & Infectious Disease Institute (VAXINFECTIO), University of Antwerp, 2610 Wilrijk, Belgium.

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. Read More

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

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

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

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

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

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

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

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

ACS Appl Nano Mater 2021 Jan 21;4(1):167-181. Epub 2020 Dec 21.

Regenerative Medicine & Cellular Therapies Division, The University of Nottingham Biodiscovery Institute (BDI), School of Pharmacy, University of Nottingham, Nottingham NG7 2RD, U.K.

Non-viral delivery systems are generally of low efficiency, which limits their use in gene therapy and editing applications. We previously developed a technology termed glycosaminoglycan (GAG)-binding enhanced transduction (GET) to efficiently deliver a variety of cargos intracellularly; our system employs GAG-binding peptides, which promote cell targeting, and cell penetrating peptides (CPPs), which enhance endocytotic cell internalization. Herein, we describe a further modification by combining gene delivery and magnetic targeting with the GET technology. Read More

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

Biomater Sci 2021 Mar 16. Epub 2021 Mar 16.

CÚRAM, SFI Research Centre for Medical Devices, National University of Ireland Galway, Ireland.

Hypertrophic scarring (HS) is an intractable complication associated with cutaneous wound healing. Although transforming growth factor β1 (TGF-β1) has long been documented as a central regulatory cytokine in fibrogenesis and fibroplasia, there is currently no cure. Gene therapy is emerging as a powerful tool to attenuate the overexpression of TGF-β1 and its signaling activities. Read More

Chem Biodivers 2021 Mar 16. Epub 2021 Mar 16.

University of KwaZulu-Natal - Westville Campus, Chemistry, University Road, Chiltern Hills, South Africa, 4000, Durban, SOUTH AFRICA.

A series of quinazolin-4-one Schiff bases were synthesized and tested in vitro for their cytotoxicity against two cancerous cell lines (MCF-7, Caco-2) and a human embryonic cell line (HEK-293) including their antibacterial evaluation against two Gram-positive and four Gram-negative bacterial strains. Most of the quinazoline-schiff bases exhibited potent cytotoxicity against Caco-2. Compound 6f with the O-butyne functional group displayed three-fold higher cytotoxic activity (IC50 = 376. Read More

J Vis Exp 2021 02 26(168). Epub 2021 Feb 26.

Experimental Ophthalmology, University of Geneva; Department of Ophthalmology, University Hospitals of Geneva.

Age-related macular degeneration (AMD) is the most frequent cause of blindness in patients >60 years, affecting ~30 million people worldwide. AMD is a multifactorial disease influenced by environmental and genetic factors, which lead to functional impairment of the retina due to retinal pigment epithelial (RPE) cell degeneration followed by photoreceptor degradation. An ideal treatment would include the transplantation of healthy RPE cells secreting neuroprotective factors to prevent RPE cell death and photoreceptor degeneration. Read More

J Nanobiotechnology 2021 Mar 8;19(1):71. Epub 2021 Mar 8.

Department of Histopathology and Morbid Anatomy, Sir Patrick Dun Translational Research Lab, St. James's Hospital, Dublin, Ireland.

Background: siRNAs hold a great potential for cancer therapy, however, poor stability in body fluids and low cellular uptake limit their use in the clinic. To enhance the bioavailability of siRNAs in tumors, novel, safe, and effective carriers are needed.

Results: Here, we developed cationic solid lipid nanoparticles (cSLNs) to carry siRNAs targeting EphA2 receptor tyrosine kinase (siEphA2), which is overexpressed in many solid tumors including prostate cancer. Read More

Stem Cell Res 2021 Feb 25;53:102251. Epub 2021 Feb 25.

Research Centre for Medical Genetics, Moscow 115522, Russian Federation.

Cystic fibrosis is one of the most common inherited diseases caused by mutations in CFTR gene, of which F508del is the most frequent. Currently, the possibility of cell therapy including genome editing is widely discussed. We generated induced pluripotent stem cells from fibroblasts obtained from a 22-year-old woman with clinically manifested and genetically proven disease by using non-viral, non-integrating RNA reprogramming vector that contains five reprogramming factors: OCT4, KLF4, SOX2, GLIS1, and c-MYC. Read More

Avicenna J Med Biotechnol 2021 Jan-Mar;13(1):2-8

Department of Advanced Sciences and Technologies, Faculty of Medicine, North Khorasan University of Medical Sciences, Bojnurd, Iran.

Background: The application of non-viral systems for delivering genes to cells is becoming a very interesting issue, especially in the treatment of neoplasms such as Breast Cancer (BC). Polymer-based non-viral systems are safe and feasible gene carriers to be used in targeted cancer therapy. gene encodes a transcription factor and is overexpressed in some cancers. Read More

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

J Control Release 2021 Mar 4;332:346-366. Epub 2021 Mar 4.

Laboratoire de Chimie de Coordination du CNRS, 205 route de Narbonne, 31077 Toulouse Cedex 4, France; Université Toulouse 118 route de Narbonne, 31077 Toulouse Cedex 4, France. Electronic address:

Nanomedicine represents a very significant contribution in current cancer treatment; in addition to surgical intervention, radiation and chemotherapeutic agents that unfortunately also kill healthy cells, inducing highly deleterious and often life-threatening side effects in the patient. Of the numerous nanoparticles used against cancer, gold nanoparticles had been developed for therapeutic applications. Inter alia, a large variety of dendrimers, i. Read More

Pharmaceutics 2021 Feb 19;13(2). Epub 2021 Feb 19.

Pharmacology Department, Faculty of Medicine, Universidad de Valencia, 46010 Valencia, Spain.

Vector design and its characterization is an area of great interest in current vaccine research. In this article, we have formulated and characterized a multicompartmental lipopolyplex, which associates multiple liposomes and polyplexes in the same complex. These particles allow the simultaneous delivery of lipid or water-soluble antigens associated with genes to the same cell, in much higher amounts than conventional lipopolyplexes. Read More

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

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

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