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Lentivirus vector therapy

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https://www.readbyqxmd.com/read/29034262/detection-of-replication-competent-lentivirus-using-a-qpcr-assay-for-vsv-g
#1
Lindsey M Skrdlant, Randall J Armstrong, Brett M Keidaisch, Mario F Lorente, David L DiGiusto
Lentiviral vectors are a common tool used to introduce new and corrected genes into cell therapy products for treatment of human diseases. Although lentiviral vectors are ideal for delivery and stable integration of genes of interest into the host cell genome, they potentially pose risks to human health, such as integration-mediated transformation and generation of a replication competent lentivirus (RCL) capable of infecting non-target cells. In consideration of the latter risk, all cell-based products modified by lentiviral vectors and intended for patient use must be tested for RCL prior to treatment of the patient...
March 16, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/29017974/vectofusin-1-a-potent-peptidic-enhancer-of-viral-gene-transfer-forms-ph-dependent-%C3%AE-helical-nanofibrils-concentrating-viral-particles
#2
Louic S Vermeer, Loic Hamon, Alicia Schirer, Michel Schoup, Jérémie Cosette, Saliha Majdoul, David Pastré, Daniel Stockholm, Nathalie Holic, Petra Hellwig, Anne Galy, David Fenard, Burkhard Bechinger
Gene transfer using lentiviral vectors has therapeutic applications spanning from monogenic and infectious diseases to cancer. Such gene therapy has to be improved by enhancing the levels of viral infection of target cells and/or reducing the amount of lentivirus for greater safety and reduced costs. Vectofusin-1, a recently developed cationic amphipathic peptide with a pronounced capacity to enhance such viral transduction, strongly promotes the entry of several retroviral pseudotypes into target cells when added to the culture medium...
October 7, 2017: Acta Biomaterialia
https://www.readbyqxmd.com/read/28982327/comparative-study-of-adeno-associated-virus-adenovirus-baculovirus-and-lentivirus-vectors-for-gene-therapy-of-the-eyes
#3
Giedrius Kalesnykas, Emmi Kokki, Laura Alasaarela, Hanna P Lesch, Timo Tuulos, Kati Kinnunen, Hannu Uusitalo, Kari Airenne, Seppo Ylä-Herttuala
Background The eye possesses unique anatomical features that make it a valuable target for gene therapy applications. Objective The aim of the current study was to compare transduction efficiency, safety and biodistribution of four viral vectors following intravitreal injection. Method Adenovirus (AdV), adeno-associated virus (AAV), baculovirus (BV) and lentivirus (LV) vectors encoding green fluorescent protein (GFP) were injected bilaterally intravitreally into adult C57BL/6OlaHsd mice. Control mice received saline...
October 3, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28970045/absence-of-replication-competent-lentivirus-in-the-clinic-analysis-of-infused-t-cell-products
#4
Kenneth Cornetta, Lisa Duffy, Cameron J Turtle, Michael Jensen, Stephen Forman, Gwendolyn Binder-Scholl, Terry Fry, Anne Chew, David G Maloney, Carl H June
Exposure to replication-competent lentivirus (RCL) is a theoretical safety concern for individuals treated with lentiviral gene therapy. For certain ex vivo gene therapy applications, including cancer immunotherapy trials, RCL detection assays are used to screen the vector product as well as the vector-transduced cells. In this study, we reviewed T cell products screened for RCL using methodology developed in the National Gene Vector Biorepository. All trials utilized third-generation lentiviral vectors produced by transient transfection...
September 12, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28962641/overexpression-of-hypoxia-inducible-factor-1-alpha-improves-immunomodulation-by-dental-mesenchymal-stem-cells
#5
Victor G Martinez, Imelda Ontoria-Oviedo, Carolina P Ricardo, Sian E Harding, Rosa Sacedon, Alberto Varas, Agustin Zapata, Pilar Sepulveda, Angeles Vicente
BACKGROUND: Human dental mesenchymal stem cells (MSCs) are considered as highly accessible and attractive MSCs for use in regenerative medicine, yet some of their features are not as well characterized as other MSCs. Hypoxia-preconditioning and hypoxia-inducible factor 1 (HIF-1) alpha overexpression significantly improves MSC therapeutics, but the mechanisms involved are not fully understood. In the present study, we characterize immunomodulatory properties of dental MSCs and determine changes in their ability to modulate adaptive and innate immune populations after HIF-1 alpha overexpression...
September 29, 2017: Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/28941328/inducing-indel-mutation-in-the-sox6-gene-by-zinc-finger-nuclease-for-gamma-reactivation-an-approach-towards-gene-therapy-of-beta-thalassemia
#6
Mehran Modares, Laleh Shariati, Zahra Hejazi, Mansoureh Shahbazi, Mohammad Amin Tabatabaiefar, Hossein Khanahmad
β-thalassemia is a common autosomal recessive disorder characterized by a deficiency in the synthesis of β-chains.Evidences show that increased HbF levels improve the symptoms in patients with β-thalassemia or sickle cell anemia. In this study, ZFN technology was applied to induce a mutation in the binding domain region of SOX6 to reactivate γ-globin expression. The sequences coding for ZFP arrays were designed and sub cloned in TDH plus as a transfer vector. The ZFN expression was confirmed using Western blot analysis...
September 23, 2017: Journal of Cellular Biochemistry
https://www.readbyqxmd.com/read/28941274/advancements-in-the-design-and-scalable-production-of-viral-gene-transfer-vectors
#7
REVIEW
David Sharon, Amine Kamen
The last 10 years have seen a rapid expansion in the use of viral gene transfer vectors, with approved therapies and late stage clinical trials underway for the treatment of genetic disorders, and multiple forms of cancer, as well as prevention of infectious diseases through vaccination. With this increased interest and widespread adoption of viral vectors by clinicians and biopharmaceutical industries, there is an imperative to engineer safer and more efficacious vectors, and develop robust, scalable and cost-effective production platforms for industrialization...
September 23, 2017: Biotechnology and Bioengineering
https://www.readbyqxmd.com/read/28895846/in%C3%A2-vivo-hematopoietic-stem-cell-transduction
#8
REVIEW
Maximilian Richter, Daniel Stone, Carol Miao, Olivier Humbert, Hans-Peter Kiem, Thalia Papayannopoulou, André Lieber
Current protocols for hematopoietic stem cell (HSC) gene therapy, involving the transplantation of ex vivo lentivirus vector-transduced HSCs into myeloablated recipients, are complex and not without risk for the patient. In vivo HSC gene therapy can be achieved by the direct modification of HSCs in the bone marrow after intraosseous injection of gene delivery vectors. A recently developed approach involves the mobilization of HSCs from the bone marrow into peripheral the blood circulation, intravenous vector injection, and re-engraftment of genetically modified HSCs in the bone marrow...
October 2017: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/28888053/mesenchymal-stem-cells-overexpressing-interleukin-35-propagate-immunosuppressive-effects-in-mice
#9
H Guo, N Zhao, H Gao, X He
To explore generation of interleukin (IL)-35-expressing mouse adipocyte-derived mesenchymal stem cells (Ad-MSCs) using lentiviral vector and their potential immunosuppressive effects in mice. Ad-MSCs were isolated and cultured in vitro and transfected with a lentivirus vector for overexpression of the therapeutic murine IL-35 gene. IL-35 expression in transfected MSCs (IL-35-MSCs) was quantified by enzyme-linked immunosorbent assay (ELISA). The lymphocytes subsets after one-way mixed lymphocyte culture and in vivo intravenous transplantation were analysed by flow cytometry to evaluate the immunosuppressive effects of IL-35-MSCs...
November 2017: Scandinavian Journal of Immunology
https://www.readbyqxmd.com/read/28884679/the-integration-of-a-macrophage-adapted-live-vaccine-strain-of-equine-infectious-anaemia-virus-eiav-in-the-horse-genome
#10
Qiang Liu, Xue-Feng Wang, Cheng Du, Yue-Zhi Lin, Jian Ma, Yu-Hong Wang, Jian-Hua Zhou, Xiaojun Wang
Integration is an important feature of retroviruses and retrovirus-based therapeutic transfection vectors. The non-primate lentivirus equine infectious anaemia virus (EIAV) primarily targets macrophages/monocytes in vivo. Investigation of the integration features of EIAVDLV121 strains, which are adapted to donkey monocyte-derived macrophages (MDMs), is of great interest. In this study, we analysed the integration features of EIAVDLV121 in equine MDMs during in vitro infection. Our previously published integration sites (IS) for EIAVFDDV13 in fetal equine dermal (FED) cells were also analysed in parallel as references...
September 7, 2017: Journal of General Virology
https://www.readbyqxmd.com/read/28869950/interleukin-10-gene-modified-dendritic-cell-induced-type-1-regulatory-t-cells-induce-transplant-tolerance-and-impede-graft-versus-host-disease-after-allogeneic-stem-cell-transplantation
#11
Jiangbo Wan, Fang Huang, Siguo Hao, Weiwei Hu, Chuanxu Liu, Wenhao Zhang, Xiaohui Deng, Linjun Chen, Liyuan Ma, Rong Tao
BACKGROUND/AIMS: Tr1 cells can induce peripheral tolerance to self- and foreign antigens, and have been developed as a therapeutic tool for the induction of tolerance to transplanted tissue. We explored the feasibility of generating Tr1 cells by using IL-10 gene-modified recipient DCs (DCLV-IL-10) to stimulate donor naive CD4+ T cells. We also investigated some biological properties of Tr1 cells. METHODS: DCLV-IL-10 were generated through DCs transduced with a lentivirus vector carrying the IL-10 gene, and Tr1 cells were produced by using DCLV-IL-10 to stimulate naive CD4+ T cells...
2017: Cellular Physiology and Biochemistry
https://www.readbyqxmd.com/read/28854814/function-and-safety-of-lentivirus-mediated-gene-transfer-for-csf2ra-deficiency
#12
Miriam Hetzel, Takuji Suzuki, Anna Rafiei Hashtchin, Paritha Arumugam, Brenna Carey, Marc Schwabbauer, Alexandra Kuhn, Johann Meyer, Axel Schambach, Johannes Van Der Loo, Thomas Moritz, Bruce C Trapnell, Nico Lachmann
Hereditary pulmonary alveolar proteinosis (hPAP) is a rare disorder of pulmonary surfactant accumulation and hypoxemic respiratory failure caused by mutations in CSF2RA (encoding the granulocyte/macrophage colony-stimulating factor [GM-CSF] receptor α-chain [CD116]), which results in reduced GM-CSF-dependent pulmonary surfactant clearance by alveolar macrophages. While no pharmacologic therapy currently exists for hPAP, it was recently demonstrated that endotracheal instillation of wild-type or gene-corrected mononuclear phagocytes (pulmonary macrophage transplantation [PMT]) results in a significant and durable therapeutic efficacy in a validated murine model of hPAP...
August 30, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28844858/mir-221-222-promote-cancer-stem-like-cell-properties-and-tumor-growth-of-breast-cancer-via-targeting-pten-and-sustained-akt-nf-%C3%AE%C2%BAb-cox-2-activation
#13
Bailong Li, Ying Lu, Lihui Yu, Xiaocui Han, Honghai Wang, Jun Mao, Jie Shen, Bo Wang, Jianwu Tang, Chunyan Li, Bo Song
MicroRNAs (miRNAs) play an important role in regulating cancer stem cell (CSC). Previous studies have shown that microRNA-221/222 (miR-221/222) cluster are involved in the propagation of breast cancer stem cell (BCSC), however, the underlying molecular mechanisms are still not fully understood. In this study, we found that miR-221/222 were overexpressed in highly aggressive breast cancer MDA-MB-231 cells, that are enriched in markers for epithelial-mesenchymal transition (EMT) and BCSCs, than in MCF-7 cells...
August 24, 2017: Chemico-biological Interactions
https://www.readbyqxmd.com/read/28842478/leucine-rich-repeat-containing-g-protein-coupled-receptor-4-facilitates-vesicular-stomatitis-virus-infection-by-binding-vesicular-stomatitis-virus-glycoprotein
#14
Na Zhang, Hongjun Huang, Binghe Tan, Yinglei Wei, Qingqing Xiong, Yan Yan, Lili Hou, Nannan Wu, Stefan Siwko, Andrea Cimarelli, Jianrong Xu, Honghui Han, Min Qian, Mingyao Liu, Bing Du
Vesicular stomatitis virus (VSV) and rabies and Chandipura viruses belong to the Rhabdovirus family. VSV is a common laboratory virus to study viral evolution and host immune responses to viral infection, and recombinant VSV-based vectors have been widely used for viral oncolysis, vaccination, and gene therapy. Although the tropism of VSV is broad, and its envelope glycoprotein G is often used for pseudotyping other viruses, the host cellular components involved in VSV infection remain unclear. Here, we demonstrate that the host protein leucine-rich repeat-containing G protein-coupled receptor 4 (Lgr4) is essential for VSV and VSV-G pseudotyped lentivirus (VSVG-LV) to infect susceptible cells...
October 6, 2017: Journal of Biological Chemistry
https://www.readbyqxmd.com/read/28830745/baculovirus-induced-recombinant-protein-expression-in-human-mesenchymal-stromal-stem-cells-a-promoter-study
#15
Gundula Sprick, Tobias Weidner, Denise Salzig, Peter Czermak
Human mesenchymal stem cells (hMSCs) are the current workhorses of regenerative medicine and gene therapy. The corresponding vectors are usually based on lentiviruses, adenoviruses, retroviruses or adeno-associated viruses, but recently they have been joined by baculoviruses, which are more widely known for their role in the development of pesticides and vaccines. Here we show that gene transfer to an immortalized human mesenchymal stroma cell line can be achieved by baculovirus transduction. We also compared the performance of five different constitutive promoters controlling GFP expression...
October 25, 2017: New Biotechnology
https://www.readbyqxmd.com/read/28817343/suppression-of-choroidal-neovascularization-in-mice-by-subretinal-delivery-of-multigenic-lentiviral-vectors-encoding-anti-angiogenic-micrornas
#16
Anne Louise Askou, Josephine Natalia Esther Benckendorff, Andreas Holmgaard, Tina Storm, Lars Aagaard, Toke Bek, Jacob Giehm Mikkelsen, Thomas Juhl Corydon
Lentivirus-based vectors have been used for the development of potent gene therapies. Here, application of a multigenic lentiviral vector (LV) producing multiple anti-angiogenic microRNAs following subretinal delivery in a laser-induced choroidal neovascularization (CNV) mouse model is presented. This versatile LV, carrying back-to-back RNApolII-driven expression cassettes, enables combined expression of microRNAs targeting vascular endothelial growth factor A (Vegfa) mRNA and fluorescent reporters. In addition, by including a vitelliform macular dystrophy 2 (VMD2) promoter, expression of microRNAs is restricted to the retinal pigment epithelial (RPE) cells...
August 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28808269/delivery-of-large-transgene-cassettes-by-foamy-virus-vector
#17
Nathan Paul Sweeney, Jinhong Meng, Hayley Patterson, Jennifer E Morgan, Myra McClure
Viral vectors are effective tools in gene therapy, but their limited packaging capacity can be restrictive. Larger clinically-relevant vectors are needed. Foamy viruses have the largest genomes among mammalian retroviruses and their vectors have shown potential for gene therapy in preclinical studies. However, the effect of vector genome size on titre has not been determined. We inserted increasing lengths of the dystrophin open reading frame in a foamy virus vector and quantified packaged vector RNA and integrated DNA...
August 14, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28763842/-effect-of-ccr1-gene-overexpression-on-the-migration-of-bone-marrow-derived-mesenchymal-stem-cells-towards-hepatocellular-carcinoma
#18
Y Gao, X L Huang, L Zhang, L Deng, A H Yin, B C Sun, S Lu
Objective: To evaluate the effect of human CCR1 (hCCR1) gene overexpression on the migration of human bone marrow-derived mesenchymal stem cells (hMSCs) towards hepatocellular carcinoma (HCC), and to examine the application prospects of MSCs as gene delivery vectors in the treatment of HCC. Methods: The hCCR1 gene was subcloned into a lentiviral vector to generate the recombinant plasmid pLV-hCCR1. The pLV-hCCR1 plasmid and two other packaging plasmids were co-transfected into 293T cells using calcium phosphate, and the virus-containing supernatant was collected...
May 20, 2017: Zhonghua Gan Zang Bing za Zhi, Zhonghua Ganzangbing Zazhi, Chinese Journal of Hepatology
https://www.readbyqxmd.com/read/28750559/suppression-of-choroidal-neovascularization-in-mice-by-subretinal-delivery-of-multigenic-lentiviral-vectors-encoding-anti-angiogenic-micrornas
#19
Anne Louise Askou, Josephine Natalia Esther Benckendorff, Andreas Holmgaard, Tina Storm, Lars Aagaard, Toke Bek, Jacob Giehm Mikkelsen, Thomas Juhl Corydon
Lentivirus-based vectors have been used for the development of potent gene therapies. Here, we present application of a multigenic lentiviral vector (LV) producing multiple anti-angiogenic microRNAs following subretinal delivery in a laser-induced choroidal neovascularization (CNV) mouse model. This versatile LV, carrying back-to-back RNApolII-driven expression cassettes, enables combined expression of microRNAs targeting vascular endothelial growth factor A (Vegfa) mRNA, and fluorescent reporters. In addition, by including a vitelliform macular dystrophy 2 (VMD2) promoter, expression of microRNAs is restricted to the retinal pigment epithelial (RPE) cells...
July 27, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28747142/development-of-the-first-world-health-organization-lentiviral-vector-standard-toward-the-production-control-and-standardization-of-lentivirus-based-gene-therapy-products
#20
Yuan Zhao, Hannah Stepto, Christian K Schneider
Gene therapy is a rapidly evolving field. So far, there have been >2,400 gene therapy products in clinical trials and four products on the market. A prerequisite for producing gene therapy products is ensuring their quality and safety. This requires appropriately controlled and standardized production and testing procedures that result in consistent safety and efficacy. Assuring the quality and safety of lentivirus-based gene therapy products in particular presents a great challenge because they are cell-based multigene products that include viral and therapeutic proteins as well as modified cells...
August 2017: Human Gene Therapy Methods
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