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Current Gene Therapy

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
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
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
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
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
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
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
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
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
Otto-Wilhelm Merten, Mauro Mezzina, Sylvain Fisson
No abstract text is available yet for this article.
2016: Current Gene Therapy
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
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
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
Nicole Schonrock, Nicky Jonkhout, John S Mattick
The human genome sequence is freely available, nearly complete and is providing a foundation of research opportunities that are overturning our current understanding of human biology. The advent of next generation sequencing has revolutionized the way we can interrogate the genome and its transcriptional products and how we analyze, diagnose, monitor and even treat human disease. Personal genetic profiles are increasing dramatically in medical value as researchers accumulate more and more knowledge about the interaction between genetic and environmental factors that contribute to the onset of common disorders...
2016: Current Gene Therapy
Jean-Baptiste Dupont
Despite the unprecedented beneficial effects of rAAV gene therapy in animal models of Duchenne muscular dystrophy (DMD), the need to inject large amounts of vector in vivo to improve phenotype raises obvious biosafety concerns. While rAAV vectors generally exhibit a good safety profile, specific pathological phenotypes such as those observed in dystrophin-deficient muscles may promote immunotoxic/genotoxic effects. Increasing the therapeutic index of rAAV in DMD muscles by reducing the effective dose could be a pivotal means of ensuring efficient clinical translation...
2016: Current Gene Therapy
Serena Scala, Lorena Leonardelli, Luca Biasco
Over the past years, clonal tracking has gained the center stage as a unique technology capable to unveil population dynamics and hierarchical relationships in vivo. We here highlighted the main open questions related to the in vivo clonal behavior of hematopoietic cells with a particular focus on hematopoietic stem and progenitor cells and T cells as main targets of cell- and gene-therapies. We walked through the current methods applied for tracing in vivo dynamics and functions of hematopoietic cells in animal models and we described the results of early studies conducted on humans...
2016: Current Gene Therapy
Marie Pierre Rols, Damijan Miklavcic
No abstract text is available yet for this article.
2016: Current Gene Therapy
Moinecha Madi, Marie-Pierre Rols, Laure Gibot
Gene electrotransfer into the skin is of particular interest for the development of medical applications including DNA vaccination, cancer treatment, wound healing or treatment of local skin disorders. However, such clinical applications are currently limited due to poor understanding of the mechanisms governing DNA electrotransfer within human tissue. Nowadays, most studies are carried out in rodent models but rodent skin varies from human skin in terms of cell composition and architecture. We used a tissue-engineering approach to study gene electrotransfer mechanisms in a human tissue context...
2016: Current Gene Therapy
Dimitrios Agas, Fabio Concetti, Melania Capitani, Giovanna Lacava, Antonio Concetti, Luigi Marchetti, Fulvio Laus, Andrea Marchegiani, Vasco Azevedo, Maria Giovanna Sabbieti, Franco Maria Venanzi
BACKGROUND: Plasmids coding protein aggregation polypeptides from different sources have been proposed as genetic adjuvants for DNA vaccines. We reported that a plasmid (pATRex), encompassing the DNA sequence for the von Willebrand A (vWA/A) domain of the Anthrax Toxin Receptor-1 (ANTXR-1, alias TEM8, Tumor Endothelial Marker 8), acts as strong immune adjuvant by inducing formation of insoluble intracellular aggregates and subsequent cell death. OBJECTIVE: In the present study we addressed the question of whether there is any substantial immunotoxicity associated with the use of self-aggregating proteins as genetic adjuvants...
2016: Current Gene Therapy
Urska Kamensek, Marie-Pierre Rols, Maja Cemazar, Muriel Golzio
BACKGROUND: Gene Electro Transfer (GET) is a promising method for therapeutic purposes. Intratumoral GET has reached clinical evaluation for antitumor treatment. An increasing number of studies suggests that antitumor effectiveness not only depends on the transfection efficiency, but also on the induction of immune responses and vascular effects that result in the nonspecific induction of cell death. Real time noninvasive optical imaging methods allow longitudinal studies of these dynamic biological processes...
2016: Current Gene Therapy
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