Multi-targeting peptides for gene carriers with high transfection efficiency

2017 ◽  
Vol 5 (40) ◽  
pp. 8035-8051 ◽  
Author(s):  
Jing Zhao ◽  
Qian Li ◽  
Xuefang Hao ◽  
Xiangkui Ren ◽  
Jintang Guo ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Transfection Efficiency ◽  
Viral Gene ◽  
High Transfection Efficiency ◽  
Gene Carriers

Non-viral gene carriers for gene therapy have been developed for many years.

scholarly journals Fabrication and Biological Activities of Plasmid DNA Gene Carrier Nanoparticles Based on Biodegradable l-Tyrosine Polyurethane

2021 ◽  
Vol 15 (1) ◽  
pp. 17
Author(s):  
Soo-Yong Park ◽  
Yang H. Yun ◽  
Bum-Joon Park ◽  
Hyung-Il Seo ◽  
Ildoo Chung
Keyword(s):  
Gene Therapy ◽  
Cellular Uptake ◽  
Plasmid Dna ◽  
Transfection Efficiency ◽  
Biological Activities ◽  
Average Diameter ◽  
Gene Carrier ◽  
Suitable Alternative ◽  
High Transfection Efficiency ◽  
Gene Carriers

Gene therapy is a suitable alternative to chemotherapy due to the complications of drug resistance and toxicity of drugs, and is also known to reduce the occurrence of cellular mutation through the use of gene carriers. In this study, gene carrier nanoparticles with minimal toxicity and high transfection efficiency were fabricated from a biocompatible and biodegradable polymer, l-tyrosine polyurethane (LTU), which was polymerized from presynthesized desaminotyrosyl tyrosine hexyl ester (DTH) and polyethylene glycol (PEG), by using double emulsion and solvent evaporation techniques, resulting in the formation of porous nanoparticles, and then used to evaluate their potential biological activities through molecular controlled release and transfection studies. To assess cellular uptake and transfection efficiency, two model drugs, fluorescently labeled bovine serum albumin (FITC-BSA) and plasmid DNA-linear polyethylenimine (LPEI) complex, were successfully encapsulated in nanoparticles, and their transfection properties and cytotoxicities were evaluated in LX2 as a normal cell and in HepG2 and MCF7 as cancer cells. The morphology and average diameter of the LTU nanoparticles were confirmed using light microscopy, transmission electron microscopy, and dynamic light scattering, while confocal microscopy was used to validate the cellular uptake of FITC-BSA-encapsulated LTU nanoparticles. Moreover, the successful cellular uptake of LTU nanoparticles encapsulated with pDNA-LPEI and the high transfection efficiency, confirmed by gel electrophoresis and X-gal assay transfection, indicated that LTU nanoparticles had excellent cell adsorption ability, facilitated gene encapsulation, and showed the sustained release tendency of genes through transfection experiments, with an optimal concentration ratio of pDNA and LPEI of 1:10. All the above characteristics are ideal for gene carriers designed to transport and release drugs into the cytoplasm, thus facilitating effective gene therapy.

DEGRADABLE POLYETHYLENIMINE DERIVATIVES AS GENE CARRIERS

Nano LIFE ◽  
2012 ◽  
Vol 02 (01) ◽  
pp. 1230004 ◽  
Author(s):  
YOU-KYOUNG KIM ◽  
QUYNH-PHUONG LUU ◽  
MOHAMMAD ARIFUL ISLAM ◽  
YUN-JAIE CHOI ◽  
CHONG-SU CHO ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Large Scale ◽  
Transfection Efficiency ◽  
Endosomal Escape ◽  
Scale Production ◽  
Nonviral Vectors ◽  
High Transfection Efficiency ◽  
Gene Carriers

Gene therapy is a treatment for inborn and acquired diseases, although the development of safe and effective gene delivery system is a great challenge to make a gene therapy a success. Viral vectors have been used in a majority of clinics because of their high transfection efficiency in vitro and in vivo. However, their use has been limited because of several drawbacks, such as induction of immune response, recombination of wild-type viruses, limitation in the size of inserted gene, and difficulty in large-scale production. Nonviral vectors have been widely proposed safe alternatives to viral vectors because they have low immunogenicity, flexibility in the size of gene to be delivered, cell targetibility, and easy scalability of production, although they have low transfection efficiency compared to viral vectors. Among nonviral vectors, polyethylenimine (PEI) has been widely used as a standard gene carriers due to its high pH-buffering capacity for endosomal escape although high-molecular-weight PEI is too toxic owing to non-degradability. Recently, many types of degradable PEI have been studied due to high transfection efficiency with lower cytotoxicity. This review explains recent progress on the development of degradable PEIs as nonviral vectors. The present paper summarizes the transfection efficiency of DNA or silencing efficiency of small interfering RNA (siRNA) based on the kinds of degradable linkage between low PEI and crosslinkers. Degradable linkages, such as ester, disulfide, imines, carbamate, amide and ketal in the degradable PEIs are covered.

scholarly journals Biodegradable Gene Carriers Containing Rigid Aromatic Linkage with Enhanced DNA Binding and Cell Uptake

Polymers ◽  
2018 ◽  
Vol 10 (10) ◽  
pp. 1080 ◽  
Author(s):  
Ju-Hui Zhang ◽  
Hui-Zhen Yang ◽  
Ji Zhang ◽  
Yan-Hong Liu ◽  
Xi He ◽  
...  
Keyword(s):  
Dna Binding ◽  
Laser Scanning ◽  
Transfection Efficiency ◽  
Laser Scanning Microscopy ◽  
Cell Uptake ◽  
Confocal Laser ◽  
Viral Gene ◽  
Cationic Polymers ◽  
Aromatic Rings ◽  
Gene Carriers

The linking and modification of low molecular weight cationic polymers (oligomers) has become an attracted strategy to construct non-viral gene carriers with good transfection efficiency and much reduced cytotoxicity. In this study, PEI 600 Da was linked by biodegradable bridges containing rigid aromatic rings. The introduction of aromatic rings enhanced the DNA-binding ability of the target polymers and also improved the stability of the formed polymer/DNA complexes. The biodegradable property and resulted DNA release were verified by enzyme stimulated gel electrophoresis experiment. These materials have lower molecular weights compared to PEI 25 kDa, but exhibited higher transfection efficiency, especially in the presence of serum. Flow cytometry and confocal laser scanning microscopy results indicate that the polymers with aromatic rings could induce higher cellular uptake. This strategy for the construction of non-viral gene vectors may be applied as an efficient and promising method for gene delivery.

scholarly journals Biodegradable PEI modified complex micelles as gene carriers with tunable gene transfection efficiency for ECs

2016 ◽  
Vol 4 (5) ◽  
pp. 997-1008 ◽  
Author(s):  
Juan Lv ◽  
Jing Yang ◽  
Xuefang Hao ◽  
Xiangkui Ren ◽  
Yakai Feng ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Vascular Grafts ◽  
Gene Carriers ◽  
Coronary Diseases ◽  
Potential Strategy ◽  
Complex Micelles

In recent years, gene therapy has evoked an increasing interest in clinical treatments of coronary diseases because it is a potential strategy to realize rapid endothelialization of artificial vascular grafts.

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 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 Targeted Protein Liposomes-Mediated AChE Gene Therapy for Effective Liver Cancer Treatment

2020 ◽  
Author(s):  
Kai Wang ◽  
Fusheng Shang ◽  
Dagui Chen ◽  
Jianpeng Jiao ◽  
Tieliu Cao ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Liver Cancer ◽  
Nude Mice ◽  
Transfection Efficiency ◽  
Tumor Therapy ◽  
Viral Gene ◽  
Therapeutic Gene ◽  
Vector Systems

Abstract The development of highly efficient non-viral gene vector systems has very important application value in the field of cancer therapy. The high protein content of proteolipids allows for high biocompatibility, low immunogenicity, and surface modification of proteins to confer more targeted drug/gene function. For the first time, this study selected transferrin, which has hepatocellular carcinoma cell targeting function, with a liposome backbone material to construct transferrin liposome (Tf-PL), and load acetylcholinesterase (AChE) therapeutic gene for in vitro and in vivo functions evaluation. The results showed that the Tf-PL transfection efficiency was higher than that of commercial Lipo 2000, low cytotoxicity and targeted ability to liver cancer SMMC-7721 cells. After tail vein injection, Tf-PL/AChE can effectively target to liver cancer, significantly inhibiting the growth of liver cancer xenografts in nude mice, prolonging the survival time of tumor-bearing nude mice, and also does not cause significant systemic toxicities. Our study provides a strategy for proteolipids targeting the transferrin receptor to carry therapeutic gene therapy for tumors. This method has strong tumor affinity and can provide an effective vector selection for precise tumor therapy.

Tuning optimum transfection of gemini surfactant–phospholipid–DNA nanoparticles by validated theoretical modeling

Nanoscale ◽  
2019 ◽  
Vol 11 (3) ◽  
pp. 1037-1046 ◽  
Author(s):  
Sattar Taheri-Araghi ◽  
Ding-Wen Chen ◽  
Mohammad Kohandel ◽  
Sivabal Sivaloganathan ◽  
Marianna Foldvari
Keyword(s):  
Mathematical Modeling ◽  
Gene Therapy ◽  
Gemini Surfactant ◽  
Transfection Efficiency ◽  
Structural Features ◽  
Coarse Grained ◽  
Viral Gene ◽  
Dna Nanoparticles ◽  
Non Invasive ◽  
Viral Gene Delivery

Coarse-grained mathematical modeling using the polymorphic structural features of gemini nanoparticles assists designing non-viral gene delivery systems with high transfection efficiency for applications in non-invasive gene therapy.

Highly Branched Poly(β-amino esters) for Non-Viral Gene Delivery: High Transfection Efficiency and Low Toxicity Achieved by Increasing Molecular Weight

2016 ◽  
Vol 17 (11) ◽  
pp. 3640-3647 ◽  
Author(s):  
Yongsheng Gao ◽  
Jian-Yuan Huang ◽  
Jonathan O’Keeffe Ahern ◽  
Lara Cutlar ◽  
Dezhong Zhou ◽  
...  
Keyword(s):  
Molecular Weight ◽  
Gene Delivery ◽  
Transfection Efficiency ◽  
Viral Gene ◽  
Amino Esters ◽  
High Transfection Efficiency ◽  
Low Toxicity ◽  
Viral Gene Delivery
Sign in / Sign up

Export Citation Format

Share Document