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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

A method for gene knockdown in the retina using a lipid-based carrier.
Conclusions: This work supports the use of Invivofectamine 3.0 as a transfection agent for effective delivery of nucleic acids to the retina for gene function studies and as potential therapeutics. PMID: 32165826 [PubMed - in process]
Source: Molecular Vision - March 15, 2020 Category: Molecular Biology Tags: Mol Vis Source Type: research

Limb Girdle Muscular Dystrophy and Therapy: Current progress in Cell and Gene-based approaches.
Abstract The limb-girdle muscular dystrophies (LGMD) are genetically heterogeneous disorders, responsible for muscle wasting and severe form of dystrophies. Despite the critical developments in the insight and information of pathomechanisms of limb-girdle muscular dystrophy, any definitive treatments do not exist, and current strategies are only based on the improvement of the signs of disorder and to enhance the life quality without resolving an underlying cause. There is a crucial relationship between pharmacological therapy and different consequences; therefore, other treatment strategies will be required. New ...
Source: Current Gene Therapy - February 16, 2020 Category: Genetics & Stem Cells Authors: Taheri F, Taghizadeh E, Pour MJR, Rostami D, Renani PG, Rastgar-Moghadam A, Hayat SMG Tags: Curr Gene Ther Source Type: research

A novel tumour suppressor lncRNA F630028O10Rik inhibits lung cancer angiogenesis by regulating miR-223-3p.
In this study, we carried out RNA sequencing (RNA-Seq) of tumour tissues isolated from LLC tumour-bearing mice treated with either Plasmodium yoelli (Py)-infected red blood cells or uninfected red blood cells. We found that F630028O10Rik (abbreviated as F63) is a novel lncRNA that was significantly up-regulated in tumours isolated from mice treated with Py-infected red blood cells compared to the control. By using gene silencing technique, F63 was found to inhibit both tumour Vascular Endothelial Growth Factor A (VEGFA) secretion and endothelial cells clone formation, migration, invasion and tube formation. Injection of ch...
Source: J Cell Mol Med - February 12, 2020 Category: Molecular Biology Authors: Qin L, Zhong M, Adah D, Qin L, Chen X, Ma C, Fu Q, Zhu X, Li Z, Wang N, Chen Y Tags: J Cell Mol Med Source Type: research

Exosomes-mediated synthetic Dicer substrates delivery for intracellular Dicer imaging detection.
In this study, we design a fluorescent labeling Dicer substrate and effectively deliver it into cell by exosomes derived from the target parent cells for intracellular Dicer expression level monitor and gene therapy. Using pre-miRNA let-7a as a model, the Dicer substrates with two terminals labeled with fluorescent and quencher group respectively was obtained by T4 RNA mediated ligase reaction from two short RNA sequences. Then, the substrate was packaged into exosomes by electroporation and delivered to target cells for intracellular dicer imaging detection. After packaging substrates into exosomes with little immunogenic...
Source: Biosensors and Bioelectronics - February 1, 2020 Category: Biotechnology Authors: Dai W, Su L, Lu H, Dong H, Zhang X Tags: Biosens Bioelectron Source Type: research

Silencing of IL-6 and STAT3 by siRNA loaded hyaluronate-N,N,N-trimethyl chitosan nanoparticles potently reduces cancer cell progression.
Abstract The immunosuppressive nature of the tumor microenvironment is a critical problem that should be considered before the design of immunotherapies. Interleukin (IL)-6 and its related downstream molecules such as signal transducer and activator of transcription (STAT)3 play an important role in the cancer progression, which can be considered as potential therapeutic targets. In the present study, we generated the active-targeted hyaluronate (HA) recoated N, N, N-trimethyl chitosan (TMC) nanoparticles (NPs) to deliver IL-6- and STAT3-specific small interfering RNAs (siRNAs) to the CD44-expressing cancer cells....
Source: International Journal of Biological Macromolecules - January 27, 2020 Category: Biochemistry Authors: Masjedi A, Ahmadi A, Atyabi F, Farhadi S, Irandoust M, Khazaei-Poul Y, Chaleshtari MG, Fathabad ME, Baghaei M, Haghnavaz N, Baradaran B, Hojjat-Farsangi M, Ghalamfarsa G, Sabz G, Hasanzadeh S, Jadidi-Niaragh F Tags: Int J Biol Macromol Source Type: research

Research Progress of nucleic acid delivery vectors for gene therapy.
Abstract Gene therapy has broad prospects as an effective treatment for some cancers and hereditary diseases. However, DNA and siRNA are easily degraded in vivo because of their biological activities as macromolecules, and they need the effective transmembrane delivery carrier Selecting the appropriate carrier for delivery will allow nucleic acid molecules to reach their site of action and enhance delivery efficiency. Currently used nucleic acid delivery vectors can be divided into two major categories: viral and non-viral vectors. Viral carrier transport efficiency is high, but there are safety issues. Non-viral ...
Source: Biomedical Microdevices - January 26, 2020 Category: Biomedical Engineering Authors: Jiao Y, Xia ZL, Ze LJ, Jing H, Xin B, Fu S Tags: Biomed Microdevices Source Type: research

Cholic acid-based mixed micelles as siRNA delivery agents for gene therapy
In this study, we demonstrate the use of lipids as co-surfactants for the preparation of mixed micelles to improve the siRNA delivery of cholic acid-based block copolymers. Poly(allyl glycidyl ether) (PAGE) and poly(ethylene glycol) (PEG) were polymerized on the surface of cholic acid to afford a star-shaped block copolymer with four arms (CA-PAGE-b-PEG)4. The allyl groups of PAGE were functionalized to bear primary or tertiary amines and folic acid was grafted onto the PEG chain end to increase cell uptake. (CA-PAGE-b-PEG)4 functionalized with either primary or tertiary amines show high siRNA complexation with close to 10...
Source: International Journal of Pharmaceutics - January 26, 2020 Category: Drugs & Pharmacology Source Type: research

Understanding the immunopathogenesis of autoimmune diseases by animal studies using gene therapy: A comprehensive review
This article will give a new perspective on understanding immunopathogenesis of autoimmune diseases not only in animals but also in human. Emerging approaches to investigate cytokine regulation through gene therapy may be a potential approach for the tailored immunomodulation of some autoimmune diseases near in the future.
Source: Autoimmunity Reviews - January 7, 2020 Category: Allergy & Immunology Source Type: research

The KLF6 splice variant KLF6-SV1 promotes proliferation and invasion of non-small cell lung cancer by up-regultating PI3K-AKT signaling pathway
Non-small cell lung cancer (NSCLC) is an aggressive type of lung malignancy. Most of the patients have poor prognosis. Increasing evidence has revealed an association between KLF6-SV1, known as an oncogenic splice variant of KLF6, and metastatic potential or poor prognosis in many cancers. We previously demonstrated the increased KLF6-SV1 expression in NSCLC samples. There was a significant association between increased expression of KLF6-SV1 with the pN and pTNM stages and poor survival in NSCLC patients. In the present study, we aimed to further investigate the functional role of KLF6-SV1 in the progression of NSCLC. SK-...
Source: Journal of Cancer - December 14, 2019 Category: Cancer & Oncology Authors: Nan Zhang, Qian-Qian Yan, Lu Lu, Jing-Bo Shao, Zhi-Gang Sun Tags: Research Paper Source Type: research

Exosomes-mediated synthetic dicer substrates delivery for intracellular dicer imaging detection
In this study, we design a fluorescent labeling Dicer substrate and effectively deliver it into cell by exosomes derived from the target parent cells for intracellular Dicer expression level monitor and gene therapy. Using pre-miRNA let-7a as a model, the Dicer substrates with two terminals labeled with fluorescent and quencher group respectively was obtained by T4 RNA mediated ligase reaction from two short RNA sequences. Then, the substrate was packaged into exosomes by electroporation and delivered to target cells for intracellular dicer imaging detection. After packaging substrates into exosomes with little immunogenic...
Source: Biosensors and Bioelectronics - December 10, 2019 Category: Biotechnology Source Type: research

Newer therapeutic approaches towards the management of diabetes mellitus: an update.
This article focused on the emerging therapeutic approaches other than the conventional pharmacological therapies, which include stem cell therapy, gene therapy, siRNA, nanotechnology and theranostics. PMID: 31663302 [PubMed - as supplied by publisher]
Source: Panminerva Medica - November 1, 2019 Category: General Medicine Tags: Panminerva Med Source Type: research

PTEN promotes intervertebral disc degeneration by regulating nuclear pulposus cell behaviors.
This article is protected by copyright. All rights reserved. PMID: 31663655 [PubMed - as supplied by publisher]
Source: Cell Biology International - October 29, 2019 Category: Cytology Authors: Xi Y, Ma J, Chen Y Tags: Cell Biol Int Source Type: research

P14. Abstract Title: Modulating Fibrinolysis using siRNA against Coagulation Factor XIII
High specificity, easy reversibility, and a half-life on the order of weeks; these are all major advantages that gene therapy has over traditional anticoagulants, add to that a strategy that weakens clots rather than preventing clotting altogether and prophylaxis of thrombosis may become much safer. Currently, anticoagulant drugs are used as a preventative measure for people susceptible to developing disease causing thrombi (which manifest as heart attacks, strokes, and pulmonary embolism), but they require frequent administration and their strict inhibition of clotting increases the risk of uncontrolled bleeding.
Source: Thrombosis Research - September 30, 2019 Category: Hematology Authors: A. Strilchuk, E. Conway, E. Pryzdial, P. Cullis, C. Kastrup Source Type: research

Proteomics evaluation of MDA-MB-231 breast cancer cells in response to RNAi-induced silencing of hPTTG
This study aims to evaluate cell proliferation and the downstream expression pattern of hPTTG1 gene at the mRNA and protein levels after specific down-regulation of hPTTG1 by siRNA.Main methodsThe human breast cancer MDA-MB-231 cell line was transfected with siRNA against hPTTG1. The mRNA and protein expression levels were examined by Real-time PCR and Western blot, respectively. The cell proliferation was assayed by MTS. To investigate the pattern of protein expression, total cellular protein was analyzed by 2D gel electrophoresis and mass spectroscopy. Subsequently, the possible biological consequences were determined by...
Source: Life Sciences - September 13, 2019 Category: Biology Source Type: research

The Application of the RNA Interference Technologies for KRAS: Current Status, Future Perspective and Associated Challenges.
Abstract KRAS is a member of the murine sarcoma virus oncogene-RAS gene family. It plays an important role in the prevention, diagnosis and treatment of tumors during tumor cell growth and angiogenesis. KRAS is the most commonly mutated oncogene in human cancers, such as the pancreatic cancers, colon cancers, and lung cancers. Detection of KRAS gene mutation is an important indicator for tracking the status of oncogenes, illuminating the developmental prognosis of various cancers, and the efficacy of radiotherapy and chemotherapy. However, the efficacy of different patients in clinical treatment is not the same. S...
Source: Current Topics in Medicinal Chemistry - August 27, 2019 Category: Chemistry Authors: Shao YT, Ma L, Zhang TH, Xu TR, Ye YC, Liu Y Tags: Curr Top Med Chem Source Type: research