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Current Gene Therapy

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https://www.readbyqxmd.com/read/28093967/anchored-lentiviral-vector-episomes-for-stem-cell-gene-therapy-in-fanconi-anemia
#1
Santhosh Chakkaramakkil Verghese, Peter Kurre
Fanconi anemia (FA) is an autosomal recessive¬, multisystem DNA repair disorder with prominent defects in hematopoietic stem cell maintenance that result in their progressive attrition and failure in early school age. Allogeneic stem cell transplantation has proved curative for patients with suitable donors. This, along with the characteristic survival advantage of phenotypically normal over non-corrected FA stem cells underscores the compelling rationale for stem cell gene therapy in FA. While integrating lentiviral vectors (LV) have become the preferred platform for genetic correction in several hematologic and immunodeficiency disorders, the residual oncogenic potential by these vectors raises concerns in FA stem cells with potentially preexisting genetic lesions...
January 13, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28067166/stem-cell-genetic-therapy-for-fanconi-anemia-a-new-hope
#2
Helmut Hanenberg, Katharina Roellecke, Constanze Wiek
Fanconi anemia (FA) is a rare inherited DNA disorder clinically characterized by congenital malformations, progressive bone marrow failure, and cancer susceptibility. Due to a strong survival advantage of spontaneously corrected 'normal' hematopoietic stem cells (HSCs) in a few patients, FA is considered a model disorder for genetic correction of autologous stem cells, where genetically corrected stem cells and their progeny have a strong in vivo selective advantage, ultimately leading to normal hematopoiesis...
January 9, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28067165/gene-therapy-in-fanconi-anemia-a-matter-of-time-safety-and-gene-transfer-tool-efficiency
#3
Verhoeyen Els, Francisco José Román Rodríguez, François-Loïc Cosset, Camille Lévy, Paula Rio
Fanconi anemia (FA) is a rare genetic syndrome characterized by progressive marrow failure. Gene therapy by infusion of FA-corrected autologous hematopoietic stem cells (HSCs) may offer a potential cure since it is a monogenetic disease with mutations in the FANC genes, coding for DNA repair enzymes (See review[1]). However, the collection of hCD34 +-cells in FA patients implies particular challenges because of the reduced numbers of progenitor cells present in their bone marrow (BM)[2] or mobilized peripheral blood[3-5]...
January 9, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28042780/modulation-of-the-type-i-interferon-response-defines-the-sensitivity-of-human-melanoma-cells-to-oncolytic-measles-virus
#4
Ferdaous Allagui, Carole Achard, Clarisse Panterne, Chantal Combredet, Nathalie Labarrière, Brigitte Dréno, Amel Benammar Elgaaied, Daniel Pouliquen, Frédéric Tangy, Jean-François Fonteneau, Marcela Grégoire, Nicolas Boisgerault
BACKGROUND: Oncolytic viruses such as live-attenuated, vaccine strains of measles virus (MV) have recently emerged as promising cancer treatments, having shown significant antitumor activity against a large variety of human tumors. OBJECTIVE: Our study aims at determining which parameters define the sensitivity of human melanoma cells to oncolytic MV infection. METHOD: We analyzed both in vitro and in vivo the oncolytic activity of MV against a panel of human melanoma cell lines established in our laboratory...
January 2, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28042779/antibody-delivery-mediated-by-recombinant-adeno-associated-virus-for-the-treatment-of-chronic-and-infectious-diseases
#5
Marc-André Robert, Rénald Gilbert, Bruno Gaillet
Monoclonal antibodies (mAbs) based-therapies are currently one of the most successful strategies to treat immune disorders, cancer and infectious diseases. Vectors derived from adeno-associated virus (AAV) are very attractive to deliver the genes coding the mAbs because they allow long-term expression thus, reducing the number of administrations. They can also penetrate biological barriers such as the blood-brain-barrier to transduce cells localized in immunoprivileged organs. Recent animal studies with AAV have demonstrated the capacity of AAV to deliver sufficient quantity of antibodies to confer an efficient immunoprotection against chronic and infectious diseases for several months to years...
January 2, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28042781/mir-21-an-oncogenic-target-mirna-for-cancer-therapy-molecular-mechanisms-and-recent-advancements-in-chemo-and-radio-resistance
#6
Sanaz Javanmardi, Mahmoud Reza Aghamaali, Samira Sadat Abolmaali, Samaneh Mohammadi, Ali Mohammad Tamaddon
In the past decade, miRNAs have been extensively attracted the scientist's attentions as tumor suppressors or oncogenes that have been implicated in tumor progression, metastasis and intrinsic resistance to various cancer therapies. microRNA-21 (miR-21) demonstrates a potential oncogenic function and target tumor inhibitor proteins in almost all types of cancer. miR-21 overexpression has been studied in terms of cell proliferation, migration, invasion, metastasis, and apoptosis regulation.Inhibition of miRNA expression using antisense technology by various nanovectors of different sizes, shapes and compositions has been evolved progressively to overcome the barriers confronted by miRNA delivery...
January 1, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/27915978/cell-compatibility-of-an-eposimal-vector-mediated-by-the-characteristic-motifs-of-matrix-attachment-regions
#7
Tian-Yun Wang, Li Wang, Yu-Xin Yang, Chun-Peng Zhao, Yan-Long Jia, Qin Li, Jun-He Zhang, Yi-You Peng, Miao Wang, Hong-Yan Xu, Xiao-Yin Wang
The characteristic sequence of β-interferon matrix attachment regions (MARs) can mediate transgene expression via episomal vectors in Chinese hamster ovary (CHO) cells. However, the host cells were from hamster ovaries, which are not suitable target cells for gene therapy. In this study, we aimed to evaluate the suitability of 12 different human cell lines as target cells for gene therapy. We transfected the cells with episomal vectors and obtained colonies stably expressing the vector products after G418 screening...
December 2, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27903222/sustained-elabela-gene-therapy-in-high-salt-induced-hypertensive-rats
#8
Claire A Schreiber, Sara J Holditch, Alex Generous, Yasuhiro Ikeda
BACKGROUND: Elabela (ELA) is a recently identified apelin receptor agonist essential for cardiac development, but its biology and therapeutic potential are unclear. In humans ELA transcripts are detected in embryonic stem cells, induced pluripotent stem cells, kidney, heart and blood vessels. ELA through the apelin receptor promotes angiogenesis in vitro, relaxes murine aortic blood vessels and attenuates high blood pressure in vivo. The apelin receptor when bound to its original ligand, apelin, exerts peripheral vasodilatory and positive inotropic effects, conferring cardioprotection in vivo...
November 21, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27903223/releasing-of-herpes-simplex-virus-carrying-ngf-in-subarachnoid-space-promotes-the-functional-repair-in-spinal-cord-injured-rats
#9
Zhi-Wei Chen, Hang-Ping Wang, Feng-Mei Yuan, Xiao Zhang, Xiu-Juan Dong, Rui-Shen Xie, Chao Tian, Bing-Shen Li, Zhen-Wu Sun, Long-Hui Zhou, Jian Liu, Tinghua Wang
BACKGROUND: Spinal cord injury is a serious disease which can lead to bad consequence in patients. Gene therapies as an effective strategy, has been developed for the treatment of disease. OBJECTIVE: Here, we explored the effect of NGF release carried by HSV-1 in the injured spinal cord. METHODS: Transgenic recombinant containing NGF was constructed by using clone technology. NGF was firstly constructed into plasimid, then enveloped by HSV-1 vector...
November 20, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27903224/evaluation-on-monocyte-mediated-delivery-of-a-therapeutic-gene-into-the-inflamed-brain
#10
Hsin-I Tong, Wen Kang, Guangzhou Zhou, Min Liu, Yingli Shi, Yuanan Lu
This study was aimed to explore the potential of a non-invasive monocytes-based delivery system to transport therapeutic genes into the diseased brain. The study was conducted by first establishing the optimized conditions for lentiviral vector (LV)-mediated gene transfer into freshly isolated monocytes, followed by investigating the inflamed-brain homing efficiency and in vivo cell-mediated transgene expression by carrier monocytes in a mouse model with acute subregional neuroinflammation. Using a newly optimized spin-infection method, up to 35% of freshly isolated monocytes were successfully transduced with the LV system DHIV-101 at M...
November 18, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27873555/different-gene-therapy-strategies-a-overview-for-prostate-cancer
#11
Aline Gomes de Souza, Victor Alexandre Felix Bastos, Isaura Beatriz Borges Silva, Karina Marangoni, Vivian Alonso Goulart
Gene therapy emerged as a mighty alternative for conventional treatment of multiple diseases. It has been defined as a product "that mediate their effects by transcription and/or translation of transferred genetic material and/or by integrating into the host genome and that are administered as nucleic acids, viruses, or genetically engineered microorganisms. The products may be used to modify cells in vivo or transferred to cells ex vivo prior to administration to the recipient". The first therapeutic gene therapy human trial was conducted in 1990 by Michael R...
November 15, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27809752/dok3-degradation-is-required-for-the-development-of-lps-induced-ards-in-mice
#12
Ning Liu, Xiaofeng Liu, Xiaoou Li, Kaifang Duan, Yuming Deng, Xiuyan Yu, Qisheng Peng
It has been reported that DOK3 protein negatively regulates LPS responses and endotoxin tolerance in mice. However, the role of DOK3 in the development of acute respiratory distress syndrome (ARDS) remains unknown. In this study, we showed that DOK3 is degraded in the lung tissues of LPS-induced ARDS. Through lentivirus infection containing DOK3(K27R) via the intranasal route, we created a mice model, in which DOK3 maintains stable expression. We found that the forced DOK3 expression significantly attenuated LPS-induced pulmonary histological alterations, inflammatory cells infiltration, lung edema, as well as the generation of inflammatory cytokines TNFα, IL-1βand IL-6 in BALF of LPS-induced ARDS mice...
November 3, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27809754/haemophilia-b-curative-fix-production-from-a-low-dose-ucoe-based-lentiviral-vector-following-hepatic-pre-natal-delivery
#13
Vincent Yu-Cheng Kao, Sonia Ferreira, Simon Nicholas Waddington, Michael Nicholas Antoniou
The ubiquitous chromatin opening element from the human HNRPA2B1-CBX3 housekeeping gene locus (A2UCOE) is able to provide stable and cell-to-cell reproducible levels of transgene expression regardless of target cell genome integration site with efficacy demonstrated in adult, embryonic and induced pluripotent stem cells and their differentiated progeny in vitro and in vivo. Here we evaluate the ability of A2UCOE-based lentiviral vectors to confer stable expression following pre-natal delivery in mice. Our results show stable post-natal A2UCOE-eGFP and A2UCOE-luciferase lentiviral vector presence in both the liver and haematopoietic system with concomitant persistence of expression demonstrating efficient transduction of both fetal hepatocytes and haematopoietic stem cells...
November 2, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27809753/immunogenicity-and-therapeutic-effects-of-pvax1-rv1419-dna-from-mycobacterium-tuberculosis
#14
Yan Liang, Xiaoyan Zhang, Li Xiao, Xuejuan Bai, Xiaomei Wang, Yourong Yang, Junxian Zhang, Jinying Song, Yinping Liu, Ning Li, Xueqiong Wu
Background The situation of tuberculosis (TB) is very severe in China. New therapeutic agents and regimens to treat TB are urgently needed. Objective In this study, a DNA vaccine expressing Mycobacterium tuberculosis (MTB) Rv1419 antigen was constructed and its immunogenicity and therapeutic effects were evaluated. Method Normal mice and TB model mice were immunized intramuscularly three times at two-week intervals with saline, plasmid vector pVAX1, M. vaccae vaccine, pVAX1- ag85a (rv3804c) DNA or pVAX1-rv1419 DNA, respectively...
November 2, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27739375/comparison-of-tetracycline-regulated-promoters-in-lentiviral-based-vectors-in-murine-transplantation-studies
#15
Maike Stahlhut, Teng-Cheong Ha, Michael Morgan, Axel Schambach, Olga S Kustikova
Tetracycline-regulated systems with efficient temporal and dose regulation of transgene expression are useful for development of new physiologic/pathophysiologic experimental models and gene therapy approaches. Lentiviral vectors with improved tetracycline-regulated promoters help to overcome the existing limitations such as basal activity in the drug absence, poor inducibility or unstable transgene expression. To compare conventional and improved tetracycline-regulated promoters in lentiviral based vectors in vivo, we investigated doxycycline-regulated gene transfer/expression levels in a long-term murine transplantation model and demonstrated that the lentiviral vector with the improved T11 promoter exhibited more efficient inducibility and higher gene transfer level...
October 13, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27487991/therapeutic-value-of-adeno-associated-virus-as-a-gene-therapy-vector-for-parkinson-s-disease-a-focused-review
#16
Aastha Singh, Dwaipayan Sen
Although adeno-associated viral vectors have been studied for a long time, its importance as a viable gene therapy strategy has been thrust into the limelight only in the recent years. Due to the admirable characteristics of these vectors, their potential has been thoughtfully utilized in the treatment of several neurodegenerative diseases. This mini-review focuses at recapitulating the therapeutic advances of adeno-associated viral vectors in the treatment of Parkinson's disease by studying the various animal model experiments and clinical trials conducted since the advent of adeno-associated viral vector - based gene therapy...
July 29, 2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27457888/editorial-thematic-issue-proceedings-from-the-embo-workshop-modern-dna-concepts-and-tools-for-safe-gene-transfer-and-modificatio
#17
EDITORIAL
Otto-Wilhelm Merten, Mauro Mezzina, Sylvain Fisson
No abstract text is available yet for this article.
2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27280971/aav-vectorization-of-dsb-mediated-gene-editing-technologies
#18
Rachel J Moser, Matthew L Hirsch
Recent work both at the bench and the bedside demonstrate zinc-finger nucleases (ZFNs), CRISPR/Cas9, and other programmable site-specific endonuclease technologies are being successfully utilized within and alongside AAV vectors to induce therapeutically relevant levels of directed gene editing within the human chromosome. Studies from past decades acknowledge that AAV vector genomes are enhanced substrates for homology-directed repair in the presence or absence of targeted DNA damage within the host genome...
2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27228988/lentiviral-delivery-of-proteins-for-genome-engineering
#19
Yujia Cai, Jacob Giehm Mikkelsen
Viruses have evolved to traverse cellular barriers and travel to the nucleus by mechanisms that involve active transport through the cytoplasm and viral quirks to resist cellular restriction factors and innate immune responses. Virus-derived vector systems exploit the capacity of viruses to ferry genetic information into cells, and now - more than three decades after the discovery of HIV - lentiviral vectors based on HIV-1 have become instrumental in biomedical research and gene therapies that require genomic insertion of transgenes...
2016: Current Gene Therapy
https://www.readbyqxmd.com/read/27216914/tet-on-systems-for-doxycycline-inducible-gene-expression
#20
Atze T Das, Liliane Tenenbaum, Ben Berkhout
The tetracycline-controlled Tet-Off and Tet-On gene expression systems are used to regulate the activity of genes in eukaryotic cells in diverse settings, varying from basic biological research to biotechnology and gene therapy applications. These systems are based on regulatory elements that control the activity of the tetracycline-resistance operon in bacteria. The Tet-Off system allows silencing of gene expression by administration of tetracycline (Tc) or tetracycline-derivatives like doxycycline (dox), whereas the Tet-On system allows activation of gene expression by dox...
2016: Current Gene Therapy
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