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Current Gene Therapy

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https://www.readbyqxmd.com/read/28403795/mesenchymal-stem-cell-derived-extracellular-vesicles-for-renal-repair
#1
Arash A Nargesi, Lilach O Lerman, Alfonso Eirin
Transplantation of autologous mesenchymal stem cells (MSCs) has been shown to attenuate renal injury and dysfunction in several animal models, and its efficacy is currently being tested in clinical trials for patients with renal disease. Accumulating evidence indicates that MSCs release extracellular vesicles (EVs) that deliver genes, microRNAs and proteins to recipient cells, acting as mediators of MSC paracrine actions. In this context, it is critical to characterize the MSC-derived EV cargo to elucidate their potential contribution to renal repair...
April 12, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28382858/new-japanese-regulatory-frameworks-for-clinical-research-and-marketing-authorization-of-gene-therapy-and-cellular-therapy-products
#2
Sumimasa Nagai, Keiya Ozawa
In Japan, the Pharmaceuticals and Medical Devices Law was passed in 2014. In this new law, regenerative medical products were defined, and a conditional and term-limited approval system only for regenerative medical products was instituted. Gene therapy and adoptive cellular therapy are categorized as regenerative medical products. This law is intended for registration trials for marketing authorization. The Act on the Safety of Regenerative Medicine was also implemented in 2014. This act is intended for clinical research and medical practice involving processed cells other than registration trials...
April 6, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28292253/the-adeno-associated-virus-a-safe-and-effective-vehicle-for-liver-specific-gene-therapy-of-inherited-and-non-inherited-diseases
#3
Kai Yan Mak, Indu G Rajapaksha, Peter W Angus, Chandana B Herath
The first human adeno-associated virus (AAV) was originally discovered in 1960s as a contaminant of adenovirus stocks preparation and thus it had not been of medical interest. Throughout last three decades AAV has gained popularity to be used in gene therapy, mainly due to its replicative defectiveness and lack of pathogenicity in human. In addition, the ability to mediate a stable and long-term expression in both non-dividing and dividing cells with specific tissue tropism makes AAV as one of the most promising candidates for therapeutic gene transfer to treat many genetic as well as non-genetic disorders...
March 14, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28215147/approaches-to-optimize-gene-therapy-for-the-treatment-of-hematologic-malignancies-overcoming-the-obstacles
#4
Yingzhe Jiang, Bing Xia, Yizhuo Zhang, Wen Xu
Gene transfer and oncolytic viruses provide new therapeutic approaches for the treatment of hematologic malignancies. However, it is still too early to introduce gene delivery or oncolytic viruses into standard clinical protocol. It is very important to discuss the obstacles that gene transfer and oncolytic virotherapy face for the further clinical application for treatment of hematologic malignancies, and updating the advances made to overcome them. The major concerns in this review include the approaches of the development of immuno-stimulatory gene transfer mediated-vaccination for leukemia therapy, RNAi-based therapy for leukemia and enhancement of sensitivity of target malignant cells to virotherapy and alteration of host immune response to favor oncolytic viruses...
February 15, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28155604/a-vector-based-on-the-chicken-hypersensitive-site-4-insulator-element-replicates-episomally-in-mammalian-cell
#5
Xi Zhang, Xiao-Yin Wang, Yan-Long Jia, Xiao Guo, Yan-Fang Wang, Tian-Yun Wang
Gene therapy in mammalian cells requires vectors exhibiting long-term stability and high expression. Episomal gene expression vectors offer a safe and attractive alternative to those that integrate into the host cell genome. In the present study, we developed a new episomal vector based on the insulator, chicken hypersensitive site 4 (cHS4). The cHS4 element was artificially synthesized, cloned into the pEGFP-C1 vector, and used to transfect Chinese hamster ovary (CHO) and human Chang liver cells. The stably transfected cell colonies were further cultured in either the presence or absence of G418 selection...
February 2, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28103787/lessons-learned-from-two-decades-of-clinical-trial-experience-in-gene-therapy-for-fanconi-anemia
#6
Jennifer E Adair, Julian Sevilla, Cristina Díaz de Heredia, Pamela S Becker, Hans-Peter Kiem, Juan Bueren
Allogeneic hematopoietic stem cell transplant is the only curative treatment for patients with the non-malignant bone marrow failure syndrome called Fanconi anemia (FA). However, early and late complications associated with this approach underscore the need for alternative treatments. Gene therapy approaches aiming to correct the genetic defect in the patient's own hematopoietic stem cells remain the most promising strategy to overcome FA-associated bone marrow failure. Yet, despite more than two decades of clinical research, a therapeutic "success" has not yet been achieved...
January 19, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28103772/induced-pluripotency-and-gene-editing-in-fanconi-anemia
#7
Susana Navarro, Alessandra Giorgetti, Angel Raya, Jakub Tolar
Induced pluripotent stem cells (iPSCs) represent an invaluable tool in a chromosomal instability syndrome such as Fanconi anemia (FA), as they can allow study of the molecular defects underlying this disease. Many other applications, such as its use as a platform to test different methods or compounds, could also be of interest. But the greatest impact of iPSCs may be in bone marrow failure diseases, as iPSCs could represent an unlimited source of autologous cells to apply in advanced treatments such as gene therapy...
January 18, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28093967/anchored-lentiviral-vector-episomes-for-stem-cell-gene-therapy-in-fanconi-anemia
#8
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
#9
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
#10
Els Verhoeyen, 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
#11
Ferdaous Allagui, Carole Achard, Clarisse Panterne, Chantal Combredet, Nathalie Labarrière, Brigitte Dréno, Amel Benammar Elgaaied, Daniel Pouliquen, Frédéric Tangy, Jean-François Fonteneau, Marc 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
#12
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
#13
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
#14
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
#15
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
#16
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
#17
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
#18
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
#19
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
#20
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
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