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Lentivirus vector therapy

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https://www.readbyqxmd.com/read/29669226/gene-therapy-in-patients-with-transfusion-dependent-%C3%AE-thalassemia
#1
Alexis A Thompson, Mark C Walters, Janet Kwiatkowski, John E J Rasko, Jean-Antoine Ribeil, Suradej Hongeng, Elisa Magrin, Gary J Schiller, Emmanuel Payen, Michaela Semeraro, Despina Moshous, Francois Lefrere, Hervé Puy, Philippe Bourget, Alessandra Magnani, Laure Caccavelli, Jean-Sébastien Diana, Felipe Suarez, Fabrice Monpoux, Valentine Brousse, Catherine Poirot, Chantal Brouzes, Jean-François Meritet, Corinne Pondarré, Yves Beuzard, Stany Chrétien, Thibaud Lefebvre, David T Teachey, Usanarat Anurathapan, P Joy Ho, Christof von Kalle, Morris Kletzel, Elliott Vichinsky, Sandeep Soni, Gabor Veres, Olivier Negre, Robert W Ross, David Davidson, Alexandria Petrusich, Laura Sandler, Mohammed Asmal, Olivier Hermine, Mariane De Montalembert, Salima Hacein-Bey-Abina, Stéphane Blanche, Philippe Leboulch, Marina Cavazzana
BACKGROUND: Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer of a marked β-globin (βA-T87Q ) gene could substitute for long-term red-cell transfusions in a patient with β-thalassemia, we wanted to evaluate the safety and efficacy of such gene therapy in patients with transfusion-dependent β-thalassemia...
April 19, 2018: New England Journal of Medicine
https://www.readbyqxmd.com/read/29599318/lentiviral-vector-mediated-gene-transfer-in-human-bladder-cancer-cell-lines
#2
Wataru Matsunaga, Misa Ichikawa, Azumi Nakamura, Takahiro Ishikawa, Akinobu Gotoh
BACKGROUND/AIM: Currently, treatment of non-muscle invasive bladder cancer causes significant deterioration in a patient's quality of life (QOL). Therefore, development of novel therapeutic options without the deterioration of QOL is very important. In this study, we assessed the anti-tumor effect of lentivirus-mediated gene transfection of tumor-suppressor genes in human bladder cancer cells. MATERIALS AND METHODS: Lentiviral vectors that contained the tumor suppressor genes, p53, p16, and PTEN, were transfected into human bladder cancer cell lines, 5637, T24, 253J, and UMUC3, and the normal human uroepithelial cell line, SV-HUC-1...
April 2018: Anticancer Research
https://www.readbyqxmd.com/read/29549248/use-of-the-xrcc2-promoter-for-in-vivo-cancer-diagnosis-and-therapy
#3
Yu Chen, Zhen Li, Zhu Xu, Huanyin Tang, Wenxuan Guo, Xiaoxiang Sun, Wenjun Zhang, Jian Zhang, Xiaoping Wan, Ying Jiang, Zhiyong Mao
The homologous recombination (HR) pathway is a promising target for cancer therapy as it is frequently upregulated in tumors. One such strategy is to target tumors with cancer-specific, hyperactive promoters of HR genes including RAD51 and RAD51C. However, the promoter size and the delivery method have limited its potential clinical applications. Here we identified the ~2.1 kb promoter of XRCC2, similar to ~6.5 kb RAD51 promoter, as also hyperactivated in cancer cells. We found that XRCC2 expression is upregulated in nearly all types of cancers, to a degree comparable to RAD51 while much higher than RAD51C...
March 16, 2018: Cell Death & Disease
https://www.readbyqxmd.com/read/29527263/homologous-recombination-mediates-stable-fah-gene-integration-and-phenotypic-correction-in-tyrosinaemia-mouse-model
#4
Norman Junge, Qinggong Yuan, Thu Huong Vu, Simon Krooss, Christien Bednarski, Asha Balakrishnan, Toni Cathomen, Michael P Manns, Ulrich Baumann, Amar Deep Sharma, Michael Ott
AIM: To stably correct tyrosinaemia in proliferating livers of fumarylacetoacetate-hydrolase knockout ( Fah -/-) mice by homologous-recombination-mediated targeted addition of the Fah gene. METHODS: C57BL/6 Fah∆exon5 mice served as an animal model for human tyrosinaemia type 1 in our study. The vector was created by amplifying human Fah cDNA including the TTR promoter from a lentivirus plasmid as described. The Fah expression cassette was flanked by homologous arms (620 bp and 749 bp long) of the Rosa26 gene locus...
February 27, 2018: World Journal of Hepatology
https://www.readbyqxmd.com/read/29467904/human-sodium-iodide-transporter-gene-mediated-imaging-and-therapy-of-mouse-glioma-comparison-between-188-re-and-131-i
#5
Rui Guo, Yun Xi, Min Zhang, Ying Miao, Miao Zhang, Biao Li
Novel treatment options are urgently required for patients with glioma who are not effectively treated through standard therapy. Human sodium iodide symporter (hNIS) is a molecular target of certain tumors types. Compared with131 I,188 Re possesses a higher energy and shorter half-life; therefore, the effects of188 Re and131 I were compared in hNIS-mediated gene imaging and therapy in the present study. Recombinant human brain glioma cell line U87 was transfected with a recombinant lentiviral vector containing hNIS (U87-hNIS)...
March 2018: Oncology Letters
https://www.readbyqxmd.com/read/29446741/what%C3%A2-s-new-in-gene-therapy-of-hemophilia
#6
E Carlos Rodriguez-Merchan
BACKGROUND: Several methods have been investigated to effectively and safely transmit genes that stimulate cells to release therapeutic factor VIII (FVIII) and factor IX (FIX) into the circulation of people with hemophilia (PWH). OBJECTIVE: To review the role of gene therapy (GT) in PWH. METHODS: A Cochrane Library and PubMed (MEDLINE) search related to the role of GT in hemophilia was analyzed. RESULTS: The most promising vectors for hemophilia GT are adeno-associated virus (AAV) and lentivirus...
February 14, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29441951/lncrna-hulc-promotes-epithelial-and-smooth-muscle-like-differentiation-of-adipose-derived-stem-cells-by-upregulation-of-bmp9
#7
Yongwei Li, Zhengfei Shan, Bin Yang, Diandong Yang, Changping Men, Yuanshan Cui, Jitao Wu
AIMS: Adipose-derived stem cells (ADSCs), a source of mesenchymal stem cells, are able to differentiate into numerous cell lineages, including epithelial and smooth muscle cells. The use of ADSCs in tissue engineering technology has become the most promising therapeutic approach for urethral reconstruction. This study aimed to explore the effect of lncRNA highly upregulated in liver cancer (HULC) on the induction of ADSCs to differentiate into epithelial and smooth-muscle-like cells. METHODS: ADSCs were isolated from a male dog, and the expression of HULC in ADSCs was overexpressed by transfection with HULC expressing vector lentivirus...
January 2, 2018: Die Pharmazie
https://www.readbyqxmd.com/read/29409356/therapeutic-potential-of-lentivirus-mediated-glucagon-like-peptide-1-glp-1-gene-therapy-for-diabetes
#8
Hale M Tasyurek, Hasan A Altunbas, Mustafa K Balci, Thomas S Griffith, Salih Sanlioglu
Postprandial glucose-induced insulin secretion from the islets of Langerhans is facilitated by glucagon-like peptide-1 (GLP-1) - a metabolic hormone with insulinotropic properties. Among the variety of effects it mediates, GLP-1 induces delta cell secretion of somatostatin, inhibits alpha cell release of glucagon, reduces gastric emptying, and slows food intake. These events collectively contribute to weight loss over time. During type 2 diabetes (T2DM), however, the incretin response to glucose is reduced and accompanied by a moderate reduction in GLP-1 secretion...
February 6, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29367346/genetically-modified-lentiviruses-that-preserve-microvascular-function-protect-against-late-radiation-damage-in-normal-tissues
#9
Aadil A Khan, James T Paget, Martin McLaughlin, Joan N Kyula, Michelle J Wilkinson, Timothy Pencavel, David Mansfield, Victoria Roulstone, Rohit Seth, Martin Halle, Navita Somaiah, Jessica K R Boult, Simon P Robinson, Hardev S Pandha, Richard G Vile, Alan A Melcher, Paul A Harris, Kevin J Harrington
Improvements in cancer survival mean that long-term toxicities, which contribute to the morbidity of cancer survivorship, are being increasingly recognized. Late adverse effects (LAEs) in normal tissues after radiotherapy (RT) are characterized by vascular dysfunction and fibrosis causing volume loss and tissue contracture, for example, in the free flaps used for immediate breast reconstruction after mastectomy. We evaluated the efficacy of lentivirally delivered superoxide dismutase 2 (SOD2) overexpression and connective tissue growth factor (CTGF) knockdown by short hairpin RNA in reducing the severity of LAEs in an animal model of free flap LAEs...
January 24, 2018: Science Translational Medicine
https://www.readbyqxmd.com/read/29361371/mass-spectrometry-analysis-reveals-differences-in-the-host-cell-protein-species-found-in-pseudotyped-lentiviral-vectors
#10
Sabine Johnson, Jun X Wheeler, Robin Thorpe, Mary Collins, Yasuhiro Takeuchi, Yuan Zhao
Lentiviral vectors (LVs) have been successfully used in clinical trials showing long term therapeutic benefits. Studying the role of cellular proteins in lentivirus HIV-1 life cycle can help understand virus assembly and budding, leading to improvement of LV production for gene therapy. Lentiviral vectors were purified using size exclusion chromatography (SEC). The cellular protein composition of LVs produced by two different methods was compared: the transient transfection system pseudotyped with the VSV-G envelope, currently used in clinical trials, and a stable producer cell system using a non-toxic envelope derived from cat endogenous retrovirus RD114, RDpro...
March 2018: Biologicals: Journal of the International Association of Biological Standardization
https://www.readbyqxmd.com/read/29276180/cd40-knocked-down-tolerogenic-dendritic-cells-decrease-diabetic-injury
#11
Aziz Mahmoudzadeh, Ali Akbar Pourfathollah, Mohammad Hossein Karimi, Seyed Mohammad Moazzeni
BACKGROUND: Type-1 diabetes (T1D) is an autoimmune disease in which T lymphocytes destroy insulin-producing β-cells. Control of self-reactive T lymphocytes and recovery of diabetic injury is the end point of T1D. OBJECTIVE: To investigate generation of tolerogenic dendritic cells (tolDCs) as an innovative method of diabetes therapy. METHODS: Lentivirus vector production was achieved by GIPZ mouse CD40 shRNA, psPAX2 and pMD2G plasmids DNA...
December 2017: Iranian Journal of Immunology: IJI
https://www.readbyqxmd.com/read/29258934/lentivirus-is-an-efficient-and-stable-transduction-vector-for-intervertebral-disc-cells
#12
Yue-Hui Zhang, Yang-Lu Zhao, Bo Li, Jia Song, Jing Zhang, Jiang Shao
OBJECTIVE: To evaluate transduction efficacy and sustainability of lentiviral vector for intervertebral disc cells both in vitro and in vivo. METHODS: Human nuclear pulposus and anulus fibrosus cells isolated from disc tissue of 28 patients during surgical disc procedures were cultured and subsequently transduced using recombinant lentivirus carrying a gene for enhanced green fluorescent protein at multiplicities of infection of 0, 15, 30, 60, 90, and 150. Cell viability was determined using the trypan blue exclusion test...
March 2018: World Neurosurgery
https://www.readbyqxmd.com/read/29246327/in-vivo-knockout-of-the-vegfa-gene-by-lentiviral-delivery-of-crispr-cas9-in-mouse-retinal-pigment-epithelium-cells
#13
Andreas Holmgaard, Anne Louise Askou, Josephine Natalia Esther Benckendorff, Emil Aagaard Thomsen, Yujia Cai, Toke Bek, Jacob Giehm Mikkelsen, Thomas J Corydon
Virus-based gene therapy by CRISPR/Cas9-mediated genome editing and knockout may provide a new option for treatment of inherited and acquired ocular diseases of the retina. In support of this notion, we show that Streptococcus pyogenes (Sp) Cas9, delivered by lentiviral vectors (LVs), can be used in vivo to selectively ablate the vascular endothelial growth factor A (Vegfa) gene in mice. By generating LVs encoding SpCas9 targeted to Vegfa, and in parallel the fluorescent eGFP marker protein, we demonstrate robust knockout of Vegfa that leads to a significant reduction of VEGFA protein in transduced cells...
December 15, 2017: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/29233896/retargeting-lentiviruses-via-spycatcher-spytag-chemistry-for-gene-delivery-into-specific-cell-types
#14
Nagarjun Kasaraneni, Ana M Chamoun-Emanuelli, Gus Wright, Zhilei Chen
We report a simple strategy for the creation of lentiviral vectors specific to any desired target cells. SpyTag is inserted into an engineered Sindbis virus envelope protein and displayed on the lentivirus surface to create Sindbis virus-SpyTag pseudoparticles (Sind-SpyTag-pp). The SpyTag serves as the covalent anchoring site for a target-cell-specific cell-binding protein (CBP) that is fused to a truncated SpyCatcher (SpyCatcherΔ). Target-cell-specific lentiviruses are created by mixing the Sind-SpyTag-pp and CBP-SpyCatcherΔ in vitro We first used a HER2-binding designed ankyrin repeat protein (DARPin...
December 12, 2017: MBio
https://www.readbyqxmd.com/read/29221804/the-impact-of-the-cd9-tetraspanin-on-lentivirus-infectivity-and-exosome-secretion
#15
Kai O Böker, Nicolas Lemus-Diaz, Rafael Rinaldi Ferreira, Lara Schiller, Stefan Schneider, Jens Gruber
Efficient transduction tools are a hallmark for both research and therapy development. Here, we introduce new insights into the generation of lentiviral vectors with improved performance by utilizing producer cells with increased production rates of extracellular vesicles through CD9 overexpression. Most human cells secrete small vesicles from their surface (microvesicles) or intraluminal endosome-derived membranes (exosomes). In particular, enhanced levels of the tetraspanin CD9 result in significantly increased numbers of extracellular vesicles with exosome-like features that were secreted from four different human cell lines...
February 7, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29212357/manufacture-of-third-generation-lentivirus-for-preclinical-use-with-process-development-considerations-for-translation-to-good-manufacturing-practice
#16
Carolina Gándara, Valerie Affleck, Elizabeth Ann Stoll
Lentiviral vectors are used in laboratories around the world for in vivo and ex vivo delivery of gene therapies, and increasingly clinical investigation as well as preclinical applications. The third-generation lentiviral vector system has many advantages, including high packaging capacity, stable gene expression in both dividing and post-mitotic cells, and low immunogenicity in the recipient organism. Yet, the manufacture of these vectors is challenging, especially at high titers required for direct use in vivo, and further challenges are presented by the process of translating preclinical gene therapies toward manufacture of products for clinical investigation...
February 2018: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/29203150/retroviral-and-lentiviral-safety-analysis-of-gene-modified-t-cell-products-and-infused-hiv-and-oncology-patients
#17
Katherine T Marcucci, Julie K Jadlowsky, Wei-Ting Hwang, Megan Suhoski-Davis, Vanessa E Gonzalez, Irina Kulikovskaya, Minnal Gupta, Simon F Lacey, Gabriela Plesa, Anne Chew, J Joseph Melenhorst, Bruce L Levine, Carl H June
Replication-competent retrovirus/lentivirus (RCR/L) and insertional oncogenesis are potential safety risks with integrating viruses in gene-modified cell therapies. As such, the Food and Drug Administration guidances outline RCR/L-monitoring methods throughout the entire gene therapy treatment cycle. We present data for 17 vector lots, 375 manufactured T cell products, and 308 patients post-infusion across both HIV and oncology indications, showing no evidence of RCR/L. Given our data, a Poisson probability model estimates that a single patient, or a group of patients, would need to be followed for at least 52...
January 3, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29188505/development-of-multigenic-lentiviral-vectors-for-cell-specific-expression-of-antiangiogenic-mirnas-and-protein-factors
#18
Anne Louise Askou, Thomas J Corydon
Generation of lentivirus (LV)-based vectors holding multiple gene cassettes for coexpression of several therapeutic factors provides potent tools in both gene delivery studies as well as in gene therapy. Here we describe the development of such multigenic LV gene delivery vectors enabling cell-specific coexpression of antiangiogenic microRNA (miRNA) and protein factors and, if preferred, a fluorescent reporter, from RNApol(II)-driven expression cassettes orientated in a back-to-back fashion. This configuration may contribute to the development of new combination therapies for amelioration of diseases involving intraocular neovascularization including exudative age-related macular degeneration (AMD)...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29179571/role-of-basal-cells-in-producing-persistent-lentivirus-mediated-airway-gene-expression
#19
Nigel Farrow, Martin Donnelley, Patricia Cmielewski, Eugene Roscioli, Nathan Rout-Pitt, Chantelle McIntyre, Ivan Bertoncello, David W Parsons
Cystic fibrosis (CF) lung disease is an ideal candidate for a genetic therapy. It has been shown previously that preconditioning with lysophosphatidylcholine (LPC) prior to lentiviral (LV) vector delivery results in long-term in vivo gene expression in the airway epithelium of CF mice. It was hypothesized that this outcome is largely due to transduction of airway basal cells that in turn pass the transgene onto their progeny. The aim of these studies was to confirm if the in vivo delivery of a human immunodeficiency virus type 1 (HIV-1) vesicular stomatitis virus envelope glycoprotein (VSV-G) pseudotyped LV vector following LPC airway conditioning results in transduction of mouse airway basal cells in situ and if the transgene is passed onto their progeny...
January 3, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29159200/analyzing-the-genotoxicity-of-retroviral-vectors-in-hematopoietic-cell-gene-therapy
#20
REVIEW
Luca Biasco, Michael Rothe, Hildegard Büning, Axel Schambach
Retroviral vectors, including those derived from gammaretroviruses and lentiviruses, have found their way into the clinical arena and demonstrated remarkable efficacy for the treatment of immunodeficiencies, leukodystrophies, and globinopathies. Despite these successes, gene therapy unfortunately also has had to face severe adverse events in the form of leukemias and myelodysplastic syndromes, related to the semi-random vector integration into the host cell genome that caused deregulation of neighboring proto-oncogenes...
March 16, 2018: Molecular Therapy. Methods & Clinical Development
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