A biodegradable adamantane polymer with ketal linkages in its backbone for gene therapy

2015 ◽  
Vol 51 (88) ◽  
pp. 15956-15959 ◽  
Author(s):  
Santanu Maity ◽  
Priya Choudhary ◽  
Manu Manjunath ◽  
Aditya Kulkarni ◽  
Niren Murthy
Keyword(s):  
Gene Therapy ◽  
Transfection Efficiency ◽  
Gene Transfection

We present a polyketal, termed pADK, which can increase the gene transfection efficiency of PEI1800–cyclodextran conjugates 60 fold and degrade into nontoxic, neutral and excretable compounds.

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.

Ultrasound molecular imaging-guided tumor gene therapy through dual-targeted cationic microbubbles

2021 ◽  
Vol 9 (7) ◽  
pp. 2454-2466
Author(s):  
Yingying Liu ◽  
Yuli Zhou ◽  
Jinfeng Xu ◽  
Hui Luo ◽  
Yao Zhu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Molecular Imaging ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Tumor Gene Therapy ◽  
Ultrasound Molecular Imaging ◽  
Tumor Gene

A novel dual-targeted cationic microbubbles help to improve gene transfection efficiency.

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 Gene Transfection of Human Turbinate Mesenchymal Stromal Cells Derived from Human Inferior Turbinate Tissues

2016 ◽  
Vol 2016 ◽  
pp. 1-10
Author(s):  
Jin Seon Kwon ◽  
Seung Hun Park ◽  
Ji Hye Baek ◽  
Truong Minh Dung ◽  
Sung Won Kim ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mesenchymal Stromal Cells ◽  
Stromal Cells ◽  
High Efficiency ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Inferior Turbinate ◽  
Nonviral Gene Delivery ◽  
Charge Ratio ◽  
Pei Nanoparticles

Human turbinate mesenchymal stromal cells (hTMSCs) are novel stem cells derived from nasal inferior turbinate tissues. They are easy to isolate from the donated tissue after turbinectomy or conchotomy. In this study, we applied hTMSCs to a nonviral gene delivery system using polyethyleneimine (PEI) as a gene carrier; furthermore, the cytotoxicity and transfection efficiency of hTMSCs were evaluated to confirm their potential as resources in gene therapy. DNA-PEI nanoparticles (NPs) were generated by adding the PEI solution to DNA and were characterized by a gel electrophoresis and by measuring particle size and surface charge of NPs. The hTMSCs were treated with DNA-PEI NPs for 4 h, and toxicity of NPs to hTMSCs and gene transfection efficiency were monitored using MTT assay, fluorescence images, and flow cytometry after 24 h and 48 h. At a high negative-to-positive charge ratio, DNA-PEI NPs treatment led to cytotoxicity of hTMSCs, but the transfection efficiency of DNA was increased due to the electrostatic effect between the NPs and the membranes of hTMSCs. Importantly, the results of this research verified that PEI could deliver DNA into hTMSCs with high efficiency, suggesting that hTMSCs could be considered as untapped resources for applications in gene therapy.

scholarly journals Injectable “nano-micron” combined gene-hydrogel microspheres for local treatment of osteoarthritis

2022 ◽  
Vol 14 (1) ◽  
Author(s):  
Bin Li ◽  
Fei Wang ◽  
Fangqiong Hu ◽  
Tao Ding ◽  
Ping Huang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Articular Cartilage ◽  
Gene Delivery ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Direct Injection ◽  
Local Treatment ◽  
Type Ii Collagen ◽  
Transfection Efficacy ◽  
Hydrogel Microspheres

AbstractSustained and controllable local gene therapy is a potential method for treating osteoarthritis (OA) through the delivery of therapeutic microRNAs (miRNAs) to targeted cells. However, direct injection of crude miRNAs for local gene therapy is limited due to its inadequate transfection efficiency, easy inactivation, and short half-life. Here, a multifunctional gene vector, arginine, histidine, and phenylalanine-modified generation 5 polyamidoamine (named G5-AHP), was employed to form G5-AHP/miR-140 nanoparticles by forming a complex with microRNA-140 (miR-140). Then, the nanoparticles were entrapped in hydrogel microspheres (MSs) to construct a “nano-micron” combined gene hydrogel to alleviate the degradation of articular cartilage. Monodisperse gelatin methacryloyl hydrogel MSs were produced under ultraviolet light using one-step innovative microfluidic technology. Evenly dispersed MSs showed better injectability in sustainable and matrix metalloproteinases (MMPs)-responsive degradation methods for local gene delivery. The G5-AHP/miR-140 nanoparticles released from the MSs exhibited high gene transfection efficacy and long-term bioactivity, facilitated endocytosis, and thus maintained the metabolic balance of cartilage matrix by promoting the expression of type II collagen and inhibiting the expression of a disintegrin and metalloproteinase with thrombospondin motifs-5 and MMP13 in chondrocytes. After injection of the “nano-micron” combined gene hydrogel into the articular cavity of the OA model, the gene hydrogel increased G5-AHP/miR-140 nanoparticle retention, prevented articular cartilage degeneration, and reduced osteophyte formation in a surgically induced mouse model of OA. The present study provides a novel cell-free approach to alleviate the progression of OA that shows potential for locally injected gene delivery systems.

PGMA-based gene carriers with lipid molecules

2016 ◽  
Vol 4 (8) ◽  
pp. 1233-1243 ◽  
Author(s):  
Chen Xu ◽  
Bingran Yu ◽  
Hao Hu ◽  
Muhammad Naeem Nizam ◽  
Wei Yuan ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Tumor Treatment ◽  
Gene Carriers ◽  
High Gene ◽  
Low Cytotoxicity

A series of effective cationic conjugations of lipid molecules with low cytotoxicity and high gene transfection efficiency were readily designed for gene therapy and tumor treatment.

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 Multifunctional Nucleus-targeting Nanoparticles with Ultra-high Gene Transfection Efficiency for In Vivo Gene Therapy

Theranostics ◽  
2017 ◽  
Vol 7 (6) ◽  
pp. 1633-1649 ◽  
Author(s):  
Ling Li ◽  
Xia Li ◽  
Yuzhe Wu ◽  
Linjiang Song ◽  
Xi Yang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
High Gene

scholarly journals Development of iRGD-Modified Peptide Carriers for Suicide Gene Therapy of Uterine Leiomyoma

Pharmaceutics ◽  
2021 ◽  
Vol 13 (2) ◽  
pp. 202
Author(s):  
Anna Egorova ◽  
Sofia Shtykalova ◽  
Alexander Selutin ◽  
Natalia Shved ◽  
Marianna Maretina ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Uterine Leiomyoma ◽  
Transfection Efficiency ◽  
Chemical Properties ◽  
Suicide Gene ◽  
Suicide Gene Therapy ◽  
Benign Tumors ◽  
Αvβ3 Integrin ◽  
Peptide Carrier ◽  
Plasmid Delivery

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.

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