Zn-promoted gene transfection efficiency for non-viral vectors: a mechanism study

2021 ◽  
Author(s):  
Xiao-Qi Yu ◽  
Rui-Mo Zhao ◽  
Yu Guo ◽  
Hui-Zhen Yang ◽  
Ji Zhang
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Viral Gene ◽  
Mechanism Study ◽  
High Affinity ◽  
Gene Vectors

The development of cationic non-viral gene vectors that may overcome the obstacles in gene delivery is of great significance to gene therapy. Metallic complexes with high affinity to nucleic acid...

scholarly journals Long-Circulating Polymeric Nanovectors for Tumor-Selective Gene Delivery

2005 ◽  
Vol 4 (6) ◽  
pp. 615-625 ◽  
Author(s):  
Sushma Kommareddy ◽  
Sandip B. Tiwari ◽  
Mansoor M. Amiji
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Viral Vectors ◽  
High Efficiency ◽  
Transfection Efficiency ◽  
The United States ◽  
Medical Intervention ◽  
Hydrophilic Polymers ◽  
Circulation Time ◽  
Viral Gene

Significant advances in the understanding of the genetic abnormalities that lead to the development, progression, and metastasis of neoplastic diseases has raised the promise of gene therapy as an approach to medical intervention. Most of the clinical protocols that have been approved in the United States for gene therapy have used the viral vectors because of the high efficiency of gene transfer. Conventional means of gene delivery using viral vectors, however, has undesirable side effects such as insertion of mutational viral gene into the host genome and development of replication competent viruses. Among non-viral gene delivery methods, polymeric nanoparticles are increasingly becoming popular as vectors of choice. The major limitation of these nanoparticles is poor transfection efficiency at the target site after systemic administration due to uptake by the cells of reticuloendothelial system (RES). In order to reduce the uptake by the cells of the RES and improve blood circulation time, these nanoparticles are coated with hydrophilic polymers such as poly(ethylene glycol) (PEG). This article reviews the use of such hydrophilic polymers employed for improving the circulation time of the nanocarriers. The mechanism of polymer coating and factors affecting the circulation time of these nanocarriers will be discussed. In addition to the long circulating property, modifications to improve the target specificity of the particles and the limitations of steric protection will be analyzed.

scholarly journals Systemic gene delivery using lipid envelope systems and its potential in overcoming challenges

2021 ◽  
pp. 46-55
Author(s):  
Miteshkumar Malaviya ◽  
Milankumar Shiroya
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Transfection Efficiency ◽  
Prolonged Release ◽  
Safety And Efficacy ◽  
Specific Targeting ◽  
Transfection Efficacy ◽  
Gene Vectors ◽  
Systemic Gene Delivery

Nonviral vectors which offer a safer and versatile alternative to viral vectors have been developed to overcome problems caused by viral carriers. However, their transfection efficacy or level of expression is substantially lower than viral vectors. Among various nonviral gene vectors, lipid envelops systems are an ideal platform for the incorporation of safety and efficacy into a single delivery system. Emerging strategies for gene delivery using lipid-based delivery systems mainly aim at improving the transfection efficiency and potency while reducing toxicity, achieving prolonged release, cell-specific targeting, co-delivery of drug and gene. Earlier efforts to improve the transfection efficiency while overcoming the toxicity led to the need for preparing conjugates of lipids with polyamines. In this review, we highlight current lipidic vectors that have been developed for gene therapy, challenges, and their solutions.

Non-Viral Vectors for Gene Delivery

2018 ◽  
Vol 9 (1) ◽  
pp. 4-11 ◽  
Author(s):  
Aparna Bansal ◽  
Himanshu
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Expression System ◽  
Target Cells ◽  
Specific Expression ◽  
Naked Dna

Introduction: Gene therapy has emerged out as a promising therapeutic pave for the treatment of genetic and acquired diseases. Gene transfection into target cells using naked DNA is a simple and safe approach which has been further improved by combining vectors or gene carriers. Both viral and non-viral approaches have achieved a milestone to establish this technique, but non-viral approaches have attained a significant attention because of their favourable properties like less immunotoxicity and biosafety, easy to produce with versatile surface modifications, etc. Literature is rich in evidences which revealed that undoubtedly, non–viral vectors have acquired a unique place in gene therapy but still there are number of challenges which are to be overcome to increase their effectiveness and prove them ideal gene vectors. Conclusion: To date, tissue specific expression, long lasting gene expression system, enhanced gene transfection efficiency has been achieved with improvement in delivery methods using non-viral vectors. This review mainly summarizes the various physical and chemical methods for gene transfer in vitro and in vivo.

scholarly journals How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?

2021 ◽  
Vol 22 (14) ◽  
pp. 7545
Author(s):  
Myriam Sainz-Ramos ◽  
Idoia Gallego ◽  
Ilia Villate-Beitia ◽  
Jon Zarate ◽  
Iván Maldonado ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Practice ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Viral Vector ◽  
Clinical Success ◽  
Genetic Material ◽  
Viral Gene ◽  
Promising Alternative ◽  
Vector Systems

Efficient delivery of genetic material into cells is a critical process to translate gene therapy into clinical practice. In this sense, the increased knowledge acquired during past years in the molecular biology and nanotechnology fields has contributed to the development of different kinds of non-viral vector systems as a promising alternative to virus-based gene delivery counterparts. Consequently, the development of non-viral vectors has gained attention, and nowadays, gene delivery mediated by these systems is considered as the cornerstone of modern gene therapy due to relevant advantages such as low toxicity, poor immunogenicity and high packing capacity. However, despite these relevant advantages, non-viral vectors have been poorly translated into clinical success. This review addresses some critical issues that need to be considered for clinical practice application of non-viral vectors in mainstream medicine, such as efficiency, biocompatibility, long-lasting effect, route of administration, design of experimental condition or commercialization process. In addition, potential strategies for overcoming main hurdles are also addressed. Overall, this review aims to raise awareness among the scientific community and help researchers gain knowledge in the design of safe and efficient non-viral gene delivery systems for clinical applications to progress in the gene therapy field.

Flexible cyclic siloxane core enhances the transfection efficiency of polyethylenimine-based non-viral gene vectors

2015 ◽  
Vol 3 (42) ◽  
pp. 8250-8267 ◽  
Author(s):  
Cristina M. Uritu ◽  
Manuela Calin ◽  
Stelian S. Maier ◽  
Corneliu Cojocaru ◽  
Alina Nicolescu ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Transfection Efficiency ◽  
Viral Gene ◽  
Cyclic Siloxane ◽  
Gene Vectors

cD4H–AGE–PEI conjugates, with a favorable balance between hydrophilic and hydrophobic moieties, are promising carriers for gene delivery.

scholarly journals The Development of Functional Non-Viral Vectors for Gene Delivery

2019 ◽  
Vol 20 (21) ◽  
pp. 5491 ◽  
Author(s):  
Patil ◽  
Gao ◽  
Lin ◽  
Li ◽  
Dang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Targeted Delivery ◽  
Viral Vectors ◽  
High Efficiency ◽  
Transfection Efficiency ◽  
Cationic Lipids ◽  
Gene Vectors ◽  
Potential Applications ◽  
Low Toxicity ◽  
Low Cytotoxicity

Gene therapy is manipulation in/of gene expression in specific cells/tissue to treat diseases. This manipulation is carried out by introducing exogenous nucleic acids, such as DNA or RNA, into the cell. Because of their negative charge and considerable larger size, the delivery of these molecules, in general, should be mediated by gene vectors. Non-viral vectors, as promising delivery systems, have received considerable attention due to their low cytotoxicity and non-immunogenicity. As research continued, more and more functional non-viral vectors have emerged. They not only have the ability to deliver a gene into the cells but also have other functions, such as the performance of fluorescence imaging, which aids in monitoring their progress, targeted delivery, and biodegradation. Recently, many reviews related to non-viral vectors, such as polymers and cationic lipids, have been reported. However, there are few reviews regarding functional non-viral vectors. This review summarizes the common functional non-viral vectors developed in the last ten years and their potential applications in the future. The transfection efficiency and the transport mechanism of these materials were also discussed in detail. We hope that this review can help researchers design more new high-efficiency and low-toxicity multifunctional non-viral vectors, and further accelerate the progress of gene therapy.

scholarly journals Non-viral Gene Therapy for Osteoarthritis

2021 ◽  
Vol 8 ◽  
Author(s):  
Ilona Uzieliene ◽  
Ursule Kalvaityte ◽  
Eiva Bernotiene ◽  
Ali Mobasheri
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Articular Chondrocytes ◽  
Transfection Efficiency ◽  
Viral Gene ◽  
Synovial Joint ◽  
Advantages And Disadvantages ◽  
Viral Gene Therapy ◽  
Viral Gene Delivery ◽  
Significant Effort

Strategies for delivering nucleic acids into damaged and diseased tissues have been divided into two major areas: viral and non-viral gene therapy. In this mini-review article we discuss the application of gene therapy for the treatment of osteoarthritis (OA), one of the most common forms of arthritis. We focus primarily on non-viral gene therapy and cell therapy. We briefly discuss the advantages and disadvantages of viral and non-viral gene therapy and review the nucleic acid transfer systems that have been used for gene delivery into articular chondrocytes in cartilage from the synovial joint. Although viral gene delivery has been more popular due to its reported efficiency, significant effort has gone into enhancing the transfection efficiency of non-viral delivery, making non-viral approaches promising tools for further application in basic, translational and clinical studies on OA. Non-viral gene delivery technologies have the potential to transform the future development of disease-modifying therapeutics for OA and related osteoarticular disorders. However, further research is needed to optimize transfection efficiency, longevity and duration of gene expression.

scholarly journals Recent Advances in the Development of Bio-Reducible Polymers for Efficient Cancer Gene Delivery Systems

2019 ◽  
Vol 2 (1) ◽  
pp. 6-13 ◽  
Author(s):  
Kiel Sung Yong ◽  
◽  
Wan Kim Sung ◽  
◽  
◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Ribonucleic Acid ◽  
Viral Vectors ◽  
Delivery Systems ◽  
Host Cells ◽  
Viral Gene ◽  
Cancer Gene ◽  
Inherited Disorders ◽  
Messenger Ribonucleic Acid

Gene therapy is the unique method for the use of genetic materials such as Messenger ribonucleic acid (mRNA), plasmid deoxyribonucleic acid (pDNA), and small interfering ribonucleic acid (siRNA) into specific host-cells for the treatment of inherited disorders in any diseases. The successful way to utilize the gene therapy is to develop the efficient cancer gene delivery systems. In this paper, the successful and efficient gene delivery systems are briefly reviewed on the basis of bio-reducible polymeric systems for cancer therapy. The viral gene delivery systems such as RNA-based viral and DNA-based viral vectors are also discussed. The development of bio-reducible polymer for gene delivery system has briefly discussed for the efficient cancer gene delivery of viral vectors and non-viral vectors.

scholarly journals Scaffold-Mediated Gene Delivery for Osteochondral Repair

Pharmaceutics ◽  
2020 ◽  
Vol 12 (10) ◽  
pp. 930 ◽  
Author(s):  
Henning Madry ◽  
Jagadeesh Kumar Venkatesan ◽  
Natalia Carballo-Pedrares ◽  
Ana Rey-Rico ◽  
Magali Cucchiarini
Keyword(s):  
Gene Delivery ◽  
Viral Vectors ◽  
Viral Gene ◽  
Therapeutic Gene ◽  
Therapeutic Concept ◽  
Osteochondral Repair ◽  
Gene Vectors ◽  
A Site

Osteochondral defects involve both the articular cartilage and the underlying subchondral bone. If left untreated, they may lead to osteoarthritis. Advanced biomaterial-guided delivery of gene vectors has recently emerged as an attractive therapeutic concept for osteochondral repair. The goal of this review is to provide an overview of the variety of biomaterials employed as nonviral or viral gene carriers for osteochondral repair approaches both in vitro and in vivo, including hydrogels, solid scaffolds, and hybrid materials. The data show that a site-specific delivery of therapeutic gene vectors in the context of acellular or cellular strategies allows for a spatial and temporal control of osteochondral neotissue composition in vitro. In vivo, implantation of acellular hydrogels loaded with nonviral or viral vectors has been reported to significantly improve osteochondral repair in translational defect models. These advances support the concept of scaffold-mediated gene delivery for osteochondral repair.

scholarly journals Novel molecular approaches to cystic fibrosis gene therapy

2005 ◽  
Vol 387 (1) ◽  
pp. 1-15 ◽  
Author(s):  
Tim W. R. LEE ◽  
David A. MATTHEWS ◽  
G. Eric BLAIR
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Bronchial Tree ◽  
Endosomal Escape ◽  
Host Cells ◽  
Cystic Fibrosis Gene ◽  
Viral Gene ◽  
Molecular Approaches

Gene therapy holds promise for the treatment of a range of inherited diseases, such as cystic fibrosis. However, efficient delivery and expression of the therapeutic transgene at levels sufficient to result in phenotypic correction of cystic fibrosis pulmonary disease has proved elusive. There are many reasons for this lack of progress, both macroscopically in terms of airway defence mechanisms and at the molecular level with regard to effective cDNA delivery. This review of approaches to cystic fibrosis gene therapy covers these areas in detail and highlights recent progress in the field. For gene therapy to be effective in patients with cystic fibrosis, the cDNA encoding the cystic fibrosis transmembrane conductance regulator protein must be delivered effectively to the nucleus of the epithelial cells lining the bronchial tree within the lungs. Expression of the transgene must be maintained at adequate levels for the lifetime of the patient, either by repeat dosage of the vector or by targeting airway stem cells. Clinical trials of gene therapy for cystic fibrosis have demonstrated proof of principle, but gene expression has been limited to 30 days at best. Results suggest that viral vectors such as adenovirus and adeno-associated virus are unsuited to repeat dosing, as the immune response reduces the effectiveness of each subsequent dose. Nonviral approaches, such as cationic liposomes, appear more suited to repeat dosing, but have been less effective. Current work regarding non-viral gene delivery is now focused on understanding the mechanisms involved in cell entry, endosomal escape and nuclear import of the transgene. There is now increasing evidence to suggest that additional ligands that facilitate endosomal escape or contain a nuclear localization signal may enhance liposome-mediated gene delivery. Much progress in this area has been informed by advances in our understanding of the mechanisms by which viruses deliver their genomes to the nuclei of host cells.

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