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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Silencing XIAP suppresses osteosarcoma cell growth, and enhances the sensitivity of osteosarcoma cells to doxorubicin and cisplatin.
Authors: Qu Y, Xia P, Zhang S, Pan S, Zhao J Abstract X-chromosome-linked inhibitor of apoptosis protein (XIAP) is an important member of the inhibitors of apoptosis (IAP) family. It has been shown that XIAP promotes the invasion, metastasis, growth and survival of malignant cells, and confers resistance to some chemotherapeutic drugs in various types of cancer. However, little is known regarding its detailed role in osteosarcoma (OS). In the present study, we first investigated the expression of XIAP in OS tissues, and an increased expression of XIAP in OS tissues compared to adjacent non-tumor tissue was ident...
Source: Oncology Reports - January 16, 2015 Category: Cancer & Oncology Tags: Oncol Rep Source Type: research

Acellular dermal matrix-based gene therapy augments graft incorporation
Acellular dermal matrix (ADM) is widely used for structural or dermal replacement purposes. Given its innate biocompatibility and its potential to vascularize, we explored the possibility of ADM to function as a small interfering RNA (siRNA) delivery system. Specifically, we sought to improve ADM vascularization by siRNA-mediated inhibition of prolyl hydroxylase domain-2 (PHD2), a cytoplasmic protein that regulates hypoxia inducible factor-1α, and improve neovascularization.
Source: Journal of Surgical Research - January 30, 2015 Category: Surgery Authors: Meredith T. Vandegrift, Caroline Spzalski, Denis Knobel, Andrew Weinstein, Maria Ham, Obinna Ezeamuzie, Stephen M. Warren, Pierre B. Saadeh Source Type: research

Analysis of UHRF1 expression in human ovarian cancer tissues and its regulation in cancer cell growth
In this study, we evaluated the expression level of UHRF1 in ovarian cancer. UHRF1 levels in paired ovarian cancer tissues and adjacent normal tissues from 80 ovarian cancer patients were detected using relative quantitatively PCR and Western blot. Small interfering RNA (siRNA) was introduced in two human ovarian cancer cell lines (SKOV-3 and OVCAR-3) to downregulate the expression of UHRF1. The proliferation of siRNA-treated cells was examined using cell counting kit-8 (CCK-8) assay. The growth of these cells showed a remarkable decrease. Moreover, flow cytometric and Hoechst 33342 assays were used to detect the apoptosis...
Source: Tumor Biology - June 13, 2015 Category: Cancer & Oncology Source Type: research

The roles of nanocarriers on pigment epithelium-derived factor in the differentiation of human cardiac stem cells
In this study, we utilized organically modified silica (ORMOSIL) nanoparticles to deliver small interfering RNA (siRNA) against pigment epithelium-derived factor (PEDF) and induce the differentiation of human cardiac stem cells (CSCs). We found that the down-regulation of PEDF can inhibit the proliferation of human CSCs and induce cell differentiation. To further study the mechanism, we have tested the Notch signalling pathway genes, Hes1 and Hes5, and found that their expressions were inhibited by the PEDF down-regulation. Furthermore, with the restoration of PEDF, both the proliferation of human CSCs and expressions of H...
Source: Cell and Tissue Research - August 12, 2015 Category: Cytology Source Type: research

Therapy for dominant inherited diseases by Allele-Specific RNA Interference: Successes and Pitfalls.
Abstract RNA interference (RNAi) is a conserved mechanism for post-transcriptional gene silencing mediated by messenger RNA (mRNA) degradation. RNAi is commonly induced by synthetic siRNA or shRNA which recognizes the targeted mRNA by base pairing and leads to target-mRNA degradation. RNAi may discriminate between two sequences only differing by one nucleotide conferring a high specificity of RNAi for its target mRNA. This property was used to develop a particular therapeutic strategy called "allele-specific-RNA interference" devoted to silence the mutated allele of genes causing dominant inherited diseases withou...
Source: Current Gene Therapy - August 12, 2015 Category: Genetics & Stem Cells Authors: Trochet D, Prudhon B, Vassilopoulos S, Bitoun M Tags: Curr Gene Ther Source Type: research

Efficient Gene Suppression in Dorsal Root Ganglia and Spinal Cord Using Adeno-Associated Virus Vectors Encoding Short-Hairpin RNA.
Abstract RNA interference is a powerful tool used to induce loss-of-function phenotypes through post-transcriptional gene silencing. Small interfering RNA (siRNA) molecules have been used to target the central nervous system (CNS) and are expected to have clinical utility against refractory neurodegenerative diseases. However, siRNA is characterized by low transduction efficiency, insufficient inhibition of gene expression, and short duration of therapeutic effects, and is thus not ideal for treatment of neural tissues and diseases. To address these problems, viral delivery of short-hairpin RNA (shRNA) expression ...
Source: Herpes - October 18, 2015 Category: Infectious Diseases Authors: Enomoto M, Hirai T, Kaburagi H, Yokota T Tags: Methods Mol Biol Source Type: research

Technological development of structural DNA/RNA-based RNAi systems and their applications.
Abstract RNA interference (RNAi)-based gene therapy has drawn tremendous attention due to its highly specific gene regulation by selective degradation of any target mRNA. There have been multiple reports regarding the development of various cationic materials for efficient siRNA delivery, however, many studies still suffer from the conventional delivery problems such as suboptimal transfection performance, a lack of tissue specificity, and potential cytotoxicity. Despite the huge therapeutic potential of siRNAs, conventional gene carriers have failed to guarantee successful gene silencing in vivo, thus not warrant...
Source: Advanced Drug Delivery Reviews - October 19, 2015 Category: Drugs & Pharmacology Authors: Jeong EH, Kim H, Jang B, Cho H, Ryu J, Kim B, Park Y, Kim J, Lee JB, Lee H Tags: Adv Drug Deliv Rev Source Type: research

Guidelines for the optimal design of miRNA-based shRNAs
Publication date: Available online 12 April 2016 Source:Methods Author(s): Xavier Bofill-De Ros, Shuo Gu RNA interference (RNAi) is an extremely useful tool for inhibiting gene expression. It can be triggered by transfected synthetic small interfering RNA (siRNA) or by expressed small hairpin RNA (shRNA). The cellular machinery processes the latter into siRNA in vivo. shRNA is preferred or required in genetic screens and specific RNAi approaches in gene therapy settings. Despite its many successes, the field of shRNAs faces many challenges. Insufficient knockdowns and off-target effects become obstacles for shRNA usage...
Source: Methods - April 14, 2016 Category: Molecular Biology Source Type: research

MDM2 knockdown mediated by a triazine-modified dendrimer in the treatment of non-small cell lung cancer.
Authors: Huang Q, Li L, Li L, Chen H, Dang Y, Zhang J, Shao N, Chang H, Zhou Z, Liu C, He B, Wei H, Xiao J Abstract Non-small cell lung cancer (NSCLC) is the most common type of lung cancer and the five-year survival rate is lower in advanced NSCLC patients. Chemotherapy is a widely used strategy in NSCLC treatment, but is usually limited by poor therapeutic efficacy and adverse effects. Therefore, a new therapeutic regimen is needed for NSCLC treatment. Gene therapy is a new strategy in the treatment of NSCLC. However, the lack of efficient and low toxic vectors remains the major obstacle. Here, we developed a bio...
Source: Oncotarget - June 5, 2016 Category: Cancer & Oncology Tags: Oncotarget Source Type: research

Mcl-1 as a potential therapeutic target for human hepatocelluar carcinoma
Summary Hepatocellular carcinoma (HCC) is a major cause of cancer-related mortality in part due to its high resistance to chemotherapeutic drugs. The anti-apoptotic Mcl-1 expression has been reported as a resistance factor in various types of tumors. Here, we investigated the expression of Mcl-1 in hepatoma cells and HCC tissues and its relationship with p53, and analyzed the possibility of the gene as a molecular target for HCC therapy. HCC specimens of 30 patients were examined by immunohistochemistry for Mcl-1 and p53 expression. Mcl-1 expression in hepatoma cell lines was measured by RT-PCR and Western blotting. The s...
Source: Journal of Huazhong University of Science and Technology -- Medical Sciences -- - July 27, 2016 Category: Research Source Type: research

From Genesis To Revelation: The Role Of Inflammatory Mediators In Chronic Respiratory Diseases And Their Control By Nucleic Acid-Based Drugs.
Abstract Asthma, chronic obstructive pulmonary disease, cystic fibrosis, and idiopathic pulmonary fibrosis, are among the most common chronic diseases and their prevalence is increasing. Each of these diseases is characterized by the secretion of cytokines and pro-inflammatory molecules which are thought to play a critical role in their pathogenesis. Moreover, immune cells, particularly neutrophils, macrophages and dendritic cells as well structural cells such as epithelial and airway smooth muscle cells are also involved in the pathogenic cycle of these diseases. There is a pressing need for the development of ne...
Source: Current Drug Delivery - August 23, 2016 Category: Drugs & Pharmacology Authors: Di Gioia S, Sardo C, Castellani S, Porsio B, Belgiovine G, Carbone A, Giammona G, Cavallaro G Tags: Curr Drug Deliv Source Type: research

Regulating gene expression towards solving ocular surface diseases
SummaryTreatment of genetic eye disease poses significant medical and surgical challenges. We used a bioluminescent corneal reporter gene mouse model to assess efficacy and potency of a number of gene therapy approaches for corneal dystrophy. Various modalities were assessed for delivery of short interfering RNA (siRNA) targeting one of five mutant alleles present in the corneal bioluminescent mouse model enabling assessment of topical, subconjunctival and intrastromal delivery. Potent and sustained in vivo gene silencing >50% for up to 7 days was observed. This siRNA therapy only provides a transient silencing of the ...
Source: Acta Ophthalmologica - September 13, 2016 Category: Opthalmology Authors: T. Moore, S. Atkinson, E. Maurizi, D. Schiroli, L. Mairs, K. Christie, I. McLean, E. Allen, D.L. Pedrioli, J. Moore, A. Nesbit Tags: Abstracts from the 2016 European Association for Vision and Eye Research Conference Source Type: research

482 Inhibition of cyclooxygenase-2 and prostaglandin E-PGE receptor 4 pathway restores ultraviolet B-induced ATP2A2/SERCA2 downregulation in keratinocytes
Background: ATP2A2 encoding the sarcoplasmic/endoplasmic reticulum Ca2+ -ATPase2 (SERCA2) is a responsible gene for Darier disease (DD). Ultraviolet (UV) B irradiation downregulates ATP2A2/SERCA2 expression in keratinocytes, whereas cyclooxygenase-2 (COX-2) expression is dramatically upregulated by UVB. Objectives: To analyze the involvement of COX-2 and subsequent Prostaglandin E (PGE) -PGE receptor signaling in ATP2A2/SERCA2 expression. Methods: Normal human keratinocytes (NHKs) were transfected with COX-2 siRNA, PGE receptor (EP1-4) siRNA and treated with COX-2 inhibitor, celecoxib, to evaluate the effect of COX-2 and P...
Source: Journal of Investigative Dermatology - April 12, 2017 Category: Dermatology Authors: M. Kaga-Kamijo, A. Wada, R. Mineki, T. Sakanishi, C. Nishiyama, S. Ikeda Tags: Genetic Disease, Gene Regulation and Gene Therapy Source Type: research

Composite liposome-PEI/nucleic acid lipopolyplexes for safe and efficient gene delivery and gene knockdown
Publication date: 1 October 2017 Source:Colloids and Surfaces B: Biointerfaces, Volume 158 Author(s): Shashank Reddy Pinnapireddy, Lili Duse, Boris Strehlow, Jens Schäfer, Udo Bakowsky Cytotoxicity is a major drawback impeding the therapeutic use of gene delivery and gene down-regulation vehicles. Apart from cytotoxicity, rapid degradation and low cellular uptake are other major factors affecting therapeutic use. Considering the above factors, formulation and development of PEI (Polyethylenimine) based, liposome encapsulated delivery vehicles with improved transfection efficiency and low cytotoxicity which can be used fo...
Source: Colloids and Surfaces B: Biointerfaces - July 4, 2017 Category: Biochemistry Source Type: research

Self ‐Assembled Aptamer‐Nanomedicine for Targeted Chemotherapy and Gene Therapy
In this study, a multifunctional aptamer‐nanomedicine (Apt‐NMed) achieving targeted chemotherapy and gene therapy of ALCL is developed. Apt‐NMed is formulated by self‐assembly of synthetic oligonucleotides containing CD30‐specific aptamer and anaplastic lymphoma kinase (ALK)‐specific siRNA followed by self‐loading of the chemotherapeutic drug doxorubicin (DOX). Apt‐NMed exhibits a well‐defined nanostructure (diameter 59 mm) and stability in human serum. Under aptamer guidance, Apt‐NMed specifically binds and internalizes targeted ALCL cells. Intracellular delivery of Apt‐NMed triggers rapid DOX releas...
Source: Small - December 4, 2017 Category: Nanotechnology Authors: Nianxi Zhao, Zihua Zeng, Youli Zu Tags: Full Paper Source Type: research