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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Peptidyl Virus ‐Like Nanovesicles as Reconfigurable “Trojan Horse” for Targeted siRNA Delivery and Synergistic Inhibition of Cancer Cells
Peptidyl virus-like nanovesicles (pVLNs) composed of bilayer membranes are assembled by the peptides and siRNA. The targeting and enzyme-responsive sequences on the bilayer's surface allow the pVLNs to enter cancer cells with high efficiency and control the release of genetic drugs in response to the subcellular environment. The resulting gene silencing efficiency is 92% and cancer cell mortality is 96%. AbstractThe self-assembly of peptidyl virus-like nanovesicles (pVLNs) composed of highly ordered peptide bilayer membranes that encapsulate the small interfering RNA (siRNA) is reported. The targeting and enzyme-responsive...
Source: Small - November 14, 2022 Category: Nanotechnology Authors: Zixuan Wang, Dongzhao Hao, Yuefei Wang, Jinwu Zhao, Jiaxing Zhang, Xi Rong, Jiaojiao Zhang, Jiwei Min, Wei Qi, Rongxin Su, Mingxia He Tags: Research Article Source Type: research

Engineered NF- κB siRNA-encapsulating exosomes as a modality for therapy of skin lesions
DiscussionOverall, this study establishes a feasible therapeutic strategy for skin injury, which may offer an alternative to conventional biological therapies requiring two or more independent compounds.
Source: Frontiers in Immunology - February 8, 2023 Category: Allergy & Immunology Source Type: research

TLR9-mediated siRNA delivery for targeting of normal and malignant human hematopoietic cells in vivo
Key Points CpG(A)-siRNA oligonucleotides allow for targeting genes specifically in human TLR9+ immune cells and blood cancer cells. Tumoricidal and immunostimulatory properties of CpG(A)-STAT3 siRNA provide a novel therapeutic opportunity for hematologic malignancies.
Source: Blood - February 21, 2013 Category: Hematology Authors: Zhang, Q., Hossain, D. M. S., Nechaev, S., Kozlowska, A., Zhang, W., Liu, Y., Kowolik, C. M., Swiderski, P., Rossi, J. J., Forman, S., Pal, S., Bhatia, R., Raubitschek, A., Yu, H., Kortylewski, M. Tags: Gene Therapy Source Type: research

Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?
Abstract RNA interference has been envisaged as a powerful tool for molecular and clinical investigation with a great potential for clinical applications. In recent years, increased understanding of cancer biology and stem cell biology has dramatically accelerated the development of technology for cell and gene therapy in these areas. This paper is a review of the most recent report of innovative use of siRNA to benefit several central nervous system diseases. Furthermore, a description is made of innovative strategies of delivery into the brain by means of viral and non-viral vectors with high potential for trans...
Source: Cellular and Molecular Life Sciences : CMLS - March 19, 2013 Category: Cytology Authors: Gherardini L, Bardi G, Gennaro M, Pizzorusso T Tags: Cell Mol Life Sci Source Type: research

Silencing of decoy receptor 3 (DcR3) expression by siRNA in pancreatic carcinoma cells induces Fas ligand-mediated apoptosis in vitro and in vivo.
In this study, we demonstrate that DcR3 is overexpressed in pancreatic carcinoma cells, and that the pancreatic carcinoma cell lines, Panc-1 and SW1990, are resistant to Fas ligand (FasL)-mediated apoptosis. To further define the function of DcR3 in cell growth and apoptosis, we used small interfering RNA (siRNA) to knockdown the expression of the DcR3 gene in Panc-1 and SW1990 cells. Our results revealed that the silencing of DcR3 expression enhanced the inhibitory effects of FasL and reduced the capabiltiy of the cells for proliferation and colony formation in vitro. In addition, the downregulation of DcR3 modulated the...
Source: International Journal of Molecular Medicine - July 11, 2013 Category: Molecular Biology Authors: Zhou J, Song S, He S, Wang Z, Zhang B, Li D, Zhu D Tags: Int J Mol Med Source Type: research

Tumor targeting RGD conjugated bio-reducible polymer for VEGF siRNA expressing plasmid delivery.
Abstract Targeted delivery of therapeutic genes to the tumor site is critical for successful and safe cancer gene therapy. The arginine grafted bio-reducible poly (cystamine bisacrylamide-diaminohexane, CBA-DAH) polymer (ABP) conjugated poly (amido amine) (PAMAM), PAM-ABP (PA) was designed previously as an efficient gene delivery carrier. To achieve high efficacy in cancer selective delivery, we developed the tumor targeting bio-reducible polymer, PA-PEG1k-RGD, by conjugating cyclic RGDfC (RGD) peptides, which bind αvβ3/5 integrins, to the PAM-ABP using polyethylene glycol (PEG, 1 kDa) as a spacer. Physical cha...
Source: Biomaterials - May 31, 2014 Category: Materials Science Authors: Kim HA, Nam K, Kim SW Tags: Biomaterials Source Type: research

SiRNA Nanotherapeutics - The Panacea of Diseases?
This article will present the major impediments that encumber successful translation of siRNA concept into reality and the ongoing research endeavours to get through those stumbling blocks along with their inadequacies. PMID: 25612905 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - January 22, 2015 Category: Genetics & Stem Cells Authors: Badrealam KF, Zubair S, Owais M Tags: Curr Gene Ther Source Type: research

Targeting Human Telomerase Reverse Transcriptase by a Simple siRNA Expression Cassette in HepG2 Cells
Conclusions: Our developed simple SEC was a powerful strategy for screening highly effective RNAi-targeted sequences and showed promise for gene therapy of HCC.
Source: Hepatitis Monthly - March 30, 2015 Category: Infectious Diseases Source Type: research

One-pot synthesis of functional poly(amino ester sulfide)s and utility in delivering pDNA and siRNA
Publication date: 18 August 2015 Source:Polymer, Volume 72 Author(s): Yunfeng Yan, Lian Xue, Jason B. Miller, Kejin Zhou, Petra Kos, Sussana Elkassih, Li Liu, Atsushi Nagai, Hu Xiong, Daniel J. Siegwart The development of efficacious carriers is an important long-standing challenge in gene therapy. In the past few decades, tremendous progress has been made toward non-viral vectors for gene delivery including cationic lipids and polymers. However, there continues to be a need for clinically translatable polymer-based delivery carriers because they offer tunable degradation profiles and functional groups, diverse...
Source: Polymer - August 16, 2015 Category: Chemistry Source Type: research

Inhibition of the effect of epidermal growth factor (EGF) on lung cancer cells. The use of plasmids encoding specific siRNA molecules
Conclusions: The inhibition of EGF system blockade with siRNA technique appears to be more effective strategy in cancer treatment in vitro, as compared to erlotinib, since this effect is probably EGFR gene mutation-independent. However, we have not definitively proved if it resulted in increased tumor cell immunogenicity.
Source: European Respiratory Journal - October 30, 2015 Category: Respiratory Medicine Authors: Gawronski, M., Kopinski, P., Jankowski, M., Goede, A., Szpechcinski, A., Chorostowska, J. Tags: 11.1 Lung Cancer Source Type: research

Silencing Bag-1 gene via magnetic gold nanoparticle-delivered siRNA plasmid for colorectal cancer therapy in vivo and in vitro
In conclusion, Bag-1 is confirmed an anti-apoptosis gene that functioned in colorectal cancer, and the mechanism of Bag-1 gene causing colorectal cancer may be related to Wnt/β-catenin signaling pathway abnormality and suggested that magnetic gold nanoparticle-delivered siRNA plasmid silencing Bag-1 is an effective gene therapy method for colorectal cancer.
Source: Tumor Biology - February 4, 2016 Category: Cancer & Oncology Source Type: research

Plasmid-Based Stat3 siRNA Delivered by Functional Graphene Oxide Suppresses Mouse Malignant Melanoma Cell Growth.
Authors: Yin D, Li Y, Guo B, Liu Z, Xu Y, Wang X, Du Y, Xu L, Meng Y, Zhao X, Zhang L Abstract RNA interference (RNAi) has been used for cancer gene therapy in recent years. However, the application of RNAi is hindered in the absence of safe and efficient gene delivery. In this article, a novel vehicle of graphene oxide functionalized with polyethylenimine and polyethylene glycol (GO-PEI-PEG) was successfully synthetized and then used to deliver plasmid-based Stat3 siRNA. The carrier can readily bind plasmid with high transfection efficiency. Moreover, molecular biology studies reveal that Stat3-related gene and pr...
Source: Oncology Research - April 23, 2016 Category: Cancer & Oncology Tags: Oncol Res Source Type: research

Modulating circulating sFlt1 in an animal model of preeclampsia using PAMAM nanoparticles for siRNA delivery
Excessive circulating sFlt1 plays a major role in the pathogenesis of preeclampsia (PE). Using RNAi to silence sFlt1 may be a therapy for treating PE. Because of the rapid degradation of siRNA, gene therapy in vivo remains limited. Poly-amidoamine (PAMAM) has been demonstrated to be an excellent nanocarrier for siRNA delivery with no discernible toxicity.
Source: Placenta - August 1, 2017 Category: Reproduction Medicine Authors: Jun Yu, Jing Jia, Xijiao Guo, Ruibao Chen, Ling Feng Source Type: research

Nanoscale polyelectrolyte complexes encapsulating mRNA and long-chained siRNA for combinatorial cancer gene therapy
Publication date: 25 August 2018 Source:Journal of Industrial and Engineering Chemistry, Volume 64 Author(s): Myung Goo Kim, Sung Duk Jo, Ji Hoon Jeong, Sun Hwa Kim To precisely regulate target genes that are abnormally expressed in cancers, we suggest an RNA-mediated multigene targeting system that co-encapsulates siRNA against vascular endothelial growth factor (VEGF) and mRNA encoding phosphatase and tensin homolog (PTEN). Polymerized long-chain siRNAs (L-siRNAs) formed stable and condensed nanocomplexes with mRNAs using thiolated glycol chitosans (tGCs) as gene carriers. The mRNA/L-siRNA/tGC nanocomplexes (MSNs) exhib...
Source: Journal of Industrial and Engineering Chemistry - June 15, 2018 Category: Chemistry Source Type: research