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Artykuły w czasopismach na temat „Lentiviral vector”

Autor: Grafiati

Data publikacji: 4 czerwca 2021

Data aktualizacji: 10 marca 2023

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1

Yew, Chee-Hong Takahiro, Narmatha Gurumoorthy, Fazlina Nordin, Gee Jun Tye, Wan Safwani Wan Kamarul Zaman, Jun Jie Tan, and Min Hwei Ng. "Integrase deficient lentiviral vector: prospects for safe clinical applications." PeerJ 10 (August 12, 2022): e13704. http://dx.doi.org/10.7717/peerj.13704.

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HIV-1 derived lentiviral vector is an efficient transporter for delivering desired genetic materials into the targeted cells among many viral vectors. Genetic material transduced by lentiviral vector is integrated into the cell genome to introduce new functions, repair defective cell metabolism, and stimulate certain cell functions. Various measures have been administered in different generations of lentiviral vector systems to reduce the vector’s replicating capabilities. Despite numerous demonstrations of an excellent safety profile of integrative lentiviral vectors, the precautionary approa

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Li, Chen, Biao Qian, Zhao Ni, Qinzhang Wang, Zixiong Wang, Luping Ma, Zhili Liu, Qiang Li, and Xinmin Wang. "Construction of recombinant lentiviral vector containing human stem cell leukemia gene and its expression in interstitial cells of cajal." Open Life Sciences 15, no. 1 (March 25, 2020): 83–91. http://dx.doi.org/10.1515/biol-2020-0010.

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AbstractThis study aims to construct recombinant lentiviral vectors containing the human stem cell leukemia (SCL) gene and investigate their in vitro transfection efficiency in Interstitial Cells of Cajal (ICC) of guinea pig bladders. In this study, the human SCL gene was successfully cloned, and the recombinant lentivirus GV287-SCL was successfully constructed. The titer of the recombinant lentivirus was 5 × 108 TU /mL. After transfecting the ICCs with the lentiviral vector at different MOIs, the optimal MOI was determined to be 10.0, and the optimal transfection time was determined to be 3 d

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Perry, Christopher, and Andrea C. M. E. Rayat. "Lentiviral Vector Bioprocessing." Viruses 13, no. 2 (February 9, 2021): 268. http://dx.doi.org/10.3390/v13020268.

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Lentiviral vectors (LVs) are potent tools for the delivery of genes of interest into mammalian cells and are now commonly utilised within the growing field of cell and gene therapy for the treatment of monogenic diseases and adoptive therapies such as chimeric antigen T-cell (CAR-T) therapy. This is a comprehensive review of the individual bioprocess operations employed in LV production. We highlight the role of envelope proteins in vector design as well as their impact on the bioprocessing of lentiviral vectors. An overview of the current state of these operations provides opportunities for b

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Wang, Nan, Narendiran Rajasekaran, Tieying Hou, and Elizabeth Mellins. "Comparison of protein expression by different lentiviral vectors (51.12)." Journal of Immunology 188, no. 1_Supplement (May 1, 2012): 51.12. http://dx.doi.org/10.4049/jimmunol.188.supp.51.12.

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Abstract HIV1-derived lentiviral vectors have been widely used as gene delivery tools due to their potent transduction capacity and stable expression after chromosomal integration in dividing and non-dividing mammalian cells. Lentiviral vectors were screened for expressing the murine class II chaperone, invariant chain (Ii), in hematopoietic stem and progenitor cells (HSPC). We compared various lentiviral vectors using GFP as a reporter gene in 293T cells and HSPC. We assessed a dual promoter vector (DP) with separate promoters for Ii and GFP, a fusion protein vector (FU) that expresses Ii and

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Nguyen, Tuan Huy, Tatiana Khakhoulina, Andrew Simmons, Philippe Morel, and Didier Trono. "A Simple and Highly Effective Method for the Stable Transduction of Uncultured Porcine Hepatocytes Using Lentiviral Vector." Cell Transplantation 14, no. 7 (August 2005): 489–96. http://dx.doi.org/10.3727/000000005783982828.

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Gene therapy is an attractive approach for the treatment of a wide spectrum of liver diseases. Lentiviral vectors allow the stable integration of transgenes into the genome of nondividing differentiated cells including hepatocytes and could provide long-lasting expression of a therapeutic gene. To develop such approaches, preclinical studies in large animal models such as pigs are necessary to evaluate the feasibility and safety of stable lentiviral integration and long-term vector expression. In addition, effective lentivector-mediated gene transfer onto porcine hepatocytes could advance in c

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6

Kubo, Shuji, and Kohnosuke Mitani. "A New Hybrid System Capable of Efficient Lentiviral Vector Production and Stable Gene Transfer Mediated by a Single Helper-Dependent Adenoviral Vector." Journal of Virology 77, no. 5 (March 1, 2003): 2964–71. http://dx.doi.org/10.1128/jvi.77.5.2964-2971.2003.

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ABSTRACT To achieve efficient and sustained gene expression, we developed a new lentivirus/adenovirus hybrid vector (LA vector) that encodes sequences required for production of a human immunodeficiency virus-based lentiviral vector (i.e., a lentiviral vector, a gag/pol/rev expression cassette, a tetracycline-inducible envelope cassette, and the tetracycline-inducible transcriptional activator cassette) in a single helper-dependent adenovirus vector backbone. Via either transfection or infection, human cell lines transduced with the LA vector produced a lentiviral vector in a doxycycline-depen

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Sakuma, Toshie, Michael A. Barry, and Yasuhiro Ikeda. "Lentiviral vectors: basic to translational." Biochemical Journal 443, no. 3 (April 16, 2012): 603–18. http://dx.doi.org/10.1042/bj20120146.

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More than two decades have passed since genetically modified HIV was used for gene delivery. Through continuous improvements these early marker gene-carrying HIVs have evolved into safer and more effective lentiviral vectors. Lentiviral vectors offer several attractive properties as gene-delivery vehicles, including: (i) sustained gene delivery through stable vector integration into host genome; (ii) the capability of infecting both dividing and non-dividing cells; (iii) broad tissue tropisms, including important gene- and cell-therapy-target cell types; (iv) no expression of viral proteins af

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8

Breckpot, Karine, David Escors, Frederick Arce, Lucienne Lopes, Katarzyna Karwacz, Sandra Van Lint, Marleen Keyaerts, and Mary Collins. "HIV-1 Lentiviral Vector Immunogenicity Is Mediated by Toll-Like Receptor 3 (TLR3) and TLR7." Journal of Virology 84, no. 11 (March 17, 2010): 5627–36. http://dx.doi.org/10.1128/jvi.00014-10.

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ABSTRACT Lentiviral vectors are promising vaccine vector candidates that have been tested extensively in preclinical models of infectious disease and cancer immunotherapy. They are also used in gene therapy clinical trials both for the ex vivo modification of cells and for direct in vivo injection. It is therefore critical to understand the mechanism(s) by which such vectors might stimulate the immune system. We evaluated the effect of lentiviral vectors on myeloid dendritic cells (DC), the main target of lentiviral transduction following subcutaneous immunization. The activation of DC culture

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Park, Frank. "Lentiviral vectors: are they the future of animal transgenesis?" Physiological Genomics 31, no. 2 (October 2007): 159–73. http://dx.doi.org/10.1152/physiolgenomics.00069.2007.

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Lentiviral vectors have become a promising new tool for the establishment of transgenic animals and the manipulation of the mammalian genome. While conventional microinjection-based methods for transgenesis have been successful in generating small and large transgenic animals, their relatively low transgenic efficiency has opened the door for alternative approaches, including lentiviral vectors. Lentiviral vectors are an appealing tool for transgenesis in part because of their ability to incorporate into genomic DNA with high efficiency, especially in cells that are not actively dividing. Lent

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Lucke, Susann, Thomas Grunwald, and Klaus Überla. "Reduced Mobilization of Rev-Responsive Element-Deficient Lentiviral Vectors." Journal of Virology 79, no. 14 (July 2005): 9359–62. http://dx.doi.org/10.1128/jvi.79.14.9359-9362.2005.

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ABSTRACT Infection of cells transduced with a lentiviral vector by human immunodeficiency virus (HIV) could lead to packaging of the lentiviral vector RNA into HIV particles and unintended transfer of the vector. To prevent this, the Rev-responsive element (RRE) of an HIV-1 vector was functionally replaced by a heterologous RNA element (MS2). Providing Rev fused to an MS2 binding protein allowed efficient vector production. Mobilization of the vector from infected target cells was below the level of detection and at least 103- to 104-fold lower than for the RRE-containing vector. Thus, RRE-def

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Joglekar, Alok V., and Salemiz Sandoval. "Pseudotyped Lentiviral Vectors: One Vector, Many Guises." Human Gene Therapy Methods 28, no. 6 (December 2017): 291–301. http://dx.doi.org/10.1089/hgtb.2017.084.

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Totsugawa, Toshinori, Naoya Kobayashi, Teru Okitsu, Hirofumi Noguchi, Takamasa Watanabe, Toshihisa Matsumura, Masanobu Maruyama, Toshiyoshi Fujiwara, Masakiyo Sakaguchi, and Noriaki Tanaka. "Lentiviral Transfer of the LacZ Gene into Human Endothelial Cells and Human Bone Marrow Mesenchymal Stem Cells." Cell Transplantation 11, no. 5 (July 2002): 481–88. http://dx.doi.org/10.3727/000000002783985620.

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Because one of the attractive characteristics of human immunodeficiency virus type 1 (HIV-1)-based lentiviral vectors is that it can infect even nondividing cells, a lentivirus-mediated gene delivery system is currently being paid a great deal of attention as an innovative tool for gene transfer into target cells. The purpose of the work was to investigate the efficacy of lentiviral transfer of the LacZ gene into human umbilical vein endothelial cells (HUVECs) and human bone marrow mesenchymal stem cells (HMSCs) in vitro. For the present study, a vesicular stomatitis virus G-protein (VSV-G)-ps

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13

Lin, Yuan, and Stanton L. Gerson. "Efficient Viral Transduction of Murine Hematopoietic Stem Cells Using HIV-1 Gag-Pol Cross Packaged Simian Immunodeficiency Viral Backbone." Blood 108, no. 11 (November 16, 2006): 5484. http://dx.doi.org/10.1182/blood.v108.11.5484.5484.

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Abstract Lentiviral vectors have been shown to infect non-dividing cells, including hematopoietic stem cell [HSC], and HIV lentiviral vector has been studied extensively in preclinical models. However low HIV lentiviral vector transduction efficiency compared to retroviral vectors, is seen in murine HSC, hampering transplantation and long-term expression of transgene in the recipients. Furthermore, concerns remain regarding the safety of HIV based vectors. Simian Immunodeficiency Viral [SIV] vectors could be safer since the parent virus does not cause disease in humans. However, to model this

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Paszkiet, Brian, Andrew Worden, Yajin Ni, Saran Bao, Franck Lemiale, Boro Dropulic, and Laurent Humeau. "CD86 and CD54 Co-Expression on VSV-G Pseudotyped HIV-1 Based Vectors Improves Transduction and Activation of Human Primary CD4+ T Lymphocytes." Blood 104, no. 11 (November 16, 2004): 1754. http://dx.doi.org/10.1182/blood.v104.11.1754.1754.

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Abstract We established the first clinical ex vivo HIV-based vector gene therapy trial in humans with HIV+ CD4+ T-cells. Briefly, this therapy involves modifying patient CD4+ T-cells with our modified lentiviral vector carrying an anti-HIV payload. These cells are then activated and expanded, and re-infused back into the patient. However, cGMP regulations require the use of costly clinical grade reagents (i.e. Retronectin™, CD3/CD28 stimulating paramagnetic beads). In an attempt to reduce ex-vivo processing costs, but not at the expense of transduction levels, we sought to determine a way to d

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Arsenijevic, Yvan, Adeline Berger, Florian Udry, and Corinne Kostic. "Lentiviral Vectors for Ocular Gene Therapy." Pharmaceutics 14, no. 8 (July 31, 2022): 1605. http://dx.doi.org/10.3390/pharmaceutics14081605.

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This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. First, the description of the global challenges encountered to develop safe and efficient lentiviral recombinant vectors for clinical application is provided. The risks and the measures taken to minimize secondary effects as well as new strategies using these vectors are also discussed. This review then focuses on lentiviral vectors specifically designed for ocular therapy and goes over preclinical and clinical studies describing their safe

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Wang, Yan, Shuai Li, Zhenyu Tian, Jiaqi Sun, Shuobin Liang, Bo Zhang, Lu Bai, et al. "Generation of a caged lentiviral vector through an unnatural amino acid for photo-switchable transduction." Nucleic Acids Research 47, no. 19 (July 30, 2019): e114-e114. http://dx.doi.org/10.1093/nar/gkz659.

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Abstract Application of viral vectors in gene delivery is attracting widespread attention but is hampered by the absence of control over transduction, which may lead to non-selective transduction with adverse side effects. To overcome some of these limitations, we proposed an unnatural amino acid aided caging–uncaging strategy for controlling the transduction capability of a viral vector. In this proof-of-principle study, we first expanded the genetic code of the lentiviral vector to incorporate an azido-containing unnatural amino acid (Nϵ-2-azidoethyloxycarbonyl-l-lysine, NAEK) site specifica

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Dyall, Julie, Jean-Baptiste Latouche, Stefan Schnell, and Michel Sadelain. "Lentivirus-transduced human monocyte-derived dendritic cells efficiently stimulate antigen-specific cytotoxic T lymphocytes." Blood 97, no. 1 (January 1, 2001): 114–21. http://dx.doi.org/10.1182/blood.v97.1.114.

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Abstract Dendritic cells (DCs) are professional antigen-presenting cells that are highly effective adjuvants for immunizing against pathogens and tumor antigens. The potential merit of genetic approaches to loading DCs with antigens is to express high and sustained levels of proteins that can be subsequently processed and presented to T lymphocytes. Replication-defective oncoretroviruses are able to efficiently transduce CD34+ progenitor-derived DCs but not monocyte-derived DCs. Here, it is shown that efficient gene transfer is obtained using a human immunodeficiency virus-1–derived lentiviral

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Chinnasamy, Nachimuthu, Michael Milsom, James Neuenfeldt, James Shaffer, Geoffrey P. Margison, Leslie J. Fairbairn, and Dhanalakshmi Chinnasamy. "Development of Novel Multigene Lentiviral Vectors." Blood 104, no. 11 (November 16, 2004): 5272. http://dx.doi.org/10.1182/blood.v104.11.5272.5272.

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Abstract Lentiviral vectors are efficient in transducing dividing and non-dividing cells. Many gene transfer applications require vectors that express more than one protein which include but not limited to therapeutic gene plus a selectable marker gene, multiple genes encoding different subunits of a complex protein or multiple independent genes that cooperate functionally. Previous strategies in constructing multigene lentiviral vectors by other groups included splicing, multiple internal promoters, internal ribosome entry site (IRES), and fusion proteins. In this study we explored the feasib

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Kim, Yoo-Jin, Yoon-Sang Kim, Andre Larochelle, Gabriel Renaud, Tyra G. Wolfsberg, Rima Adler, Robert E. Donahue, et al. "Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector–transduced CD34+ cells." Blood 113, no. 22 (May 28, 2009): 5434–43. http://dx.doi.org/10.1182/blood-2008-10-185199.

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Abstract We previously reported that lentiviral vectors derived from the simian immunodeficiency virus (SIV) were efficient at transducing rhesus hematopoietic repopulating cells. To evaluate the persistence of vector-containing and -expressing cells long term, and the safety implications of SIV lentiviral vector–mediated gene transfer, we followed 3 rhesus macaques for more than 4 years after transplantation with transduced CD34+ cells. All 3 animals demonstrated significant vector marking and expression of the GFP transgene in T cells, B cells, and granulocytes, with mean GFP+ levels of 6.7%

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Chan, Lucas, Darren Nesbeth, Taylor MacKey, Joanna Galea-Lauri, Joop Gäken, Francisco Martin, Mary Collins, Ghulam Mufti, Farzin Farzaneh, and David Darling. "Conjugation of Lentivirus to Paramagnetic Particles via Nonviral Proteins Allows Efficient Concentration and Infection of Primary Acute Myeloid Leukemia Cells." Journal of Virology 79, no. 20 (October 15, 2005): 13190–94. http://dx.doi.org/10.1128/jvi.79.20.13190-13194.2005.

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ABSTRACT Nonviral producer cell proteins incorporated into retroviral vector surfaces profoundly influence infectivity and in vivo half-life. We report the purification and concentration of lentiviral vectors using these surface proteins as an efficient gene transduction strategy. Biotinylation of these proteins and streptavidin paramagnetic particle concentration enhances titer 400- to 2,500-fold (to 109 CFU/ml for vesicular stomatitis virus G protein and 5 × 108 for amphotropic murine leukemia virus envelope). This method also uses newly introduced membrane proteins (B7.1 and ΔLNGFR) directe

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Panigaj, Martin, Michael P. Marino, and Jakob Reiser. "Tagging and Capturing of Lentiviral Vectors Using Short RNAs." International Journal of Molecular Sciences 22, no. 19 (September 23, 2021): 10263. http://dx.doi.org/10.3390/ijms221910263.

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Lentiviral (LV) vectors have emerged as powerful tools for transgene delivery ex vivo but in vivo gene therapy applications involving LV vectors have faced a number of challenges, including the low efficiency of transgene delivery, a lack of tissue specificity, immunogenicity to both the product encoded by the transgene and the vector, and the inactivation of the vector by the human complement cascade. To mitigate these issues, several engineering approaches, involving the covalent modification of vector particles or the incorporation of specific protein domains into the vector’s envelope, hav

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Liu, Shan-Lu, Christine L. Halbert, and A. Dusty Miller. "Jaagsiekte Sheep Retrovirus Envelope Efficiently Pseudotypes Human Immunodeficiency Virus Type 1-Based Lentiviral Vectors." Journal of Virology 78, no. 5 (March 1, 2004): 2642–47. http://dx.doi.org/10.1128/jvi.78.5.2642-2647.2003.

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ABSTRACT Jaagsiekte sheep retrovirus (JSRV) infects lung epithelial cells in sheep, and oncoretroviral vectors bearing JSRV Env can mediate transduction of human cells, suggesting that such vectors might be useful for lung-directed gene therapy. Here we show that JSRV Env can also efficiently pseudotype a human immunodeficiency virus type 1-based lentiviral vector, a more suitable vector for transduction of slowly dividing lung epithelial cells. We created several chimeric Env proteins that, unlike the parental Env, do not transform rodent fibroblasts but are still capable of pseudotyping lent

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Thrasher, Adrian J. "Progress in Lentiviral Vector Technologies." Human Gene Therapy 24, no. 2 (February 2013): 117–18. http://dx.doi.org/10.1089/hum.2013.2500.

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Gina Vitale. "Lentiviral vector CDMO gets funding." C&EN Global Enterprise 101, no. 5 (February 6, 2023): 13. http://dx.doi.org/10.1021/cen-10105-buscon15.

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Toon, Kamilla, Emma M. Bentley, and Giada Mattiuzzo. "More Than Just Gene Therapy Vectors: Lentiviral Vector Pseudotypes for Serological Investigation." Viruses 13, no. 2 (January 31, 2021): 217. http://dx.doi.org/10.3390/v13020217.

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Serological assays detecting neutralising antibodies are important for determining the immune responses following infection or vaccination and are also often considered a correlate of protection. The target of neutralising antibodies is usually located in the Envelope protein on the viral surface, which mediates cell entry. As such, presentation of the Envelope protein on a lentiviral particle represents a convenient alternative to handling of a potentially high containment virus or for those viruses with no established cell culture system. The flexibility, relative safety and, in most cases,

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Fung, Hua, Andrew E. Sloan, Jane Reese, Basem M. William, Karen Lingas, Lisa R. Rogers, Heather Embree, et al. "Viral Insertion Safety in Patients with Glioblastoma Who Received a Novel Lentiviral MGMT-P140K Gene Therapy to Protect Bone Marrow from Chemotherapy: No Dominant Clonal Evolution Observed with Chemo-Selection." Blood 124, no. 21 (December 6, 2014): 4801. http://dx.doi.org/10.1182/blood.v124.21.4801.4801.

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Abstract INTRODUCTION: To protect normal bone marrow from chemotherapy in glioblastoma patients, we have developed a novel strategy by introducing a strong DNA repair protein, mutant (P140K) of human methylguanine methyltransferase (MGMT), into patients’ CD34+ hematopoietic progenitors (HPC) by lentiviral gene transfer leading to selective expansion of drug-resistant P140K-MGMT CD34+ cells and their myeloid and immune cell progeny. METHODS: To achieve long-term stable expression of the P140K-MGMT gene, we used a lentiviral vector which integrates into the host genome. However, viral insertion

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Grund, Nadja, Patrick Maier, Uwe Appelt, Heike Allgayer, Frederik Wenz, W. Jens Zeller, Stefan Fruehauf, and Stefanie Laufs. "Impact of Chemoselective Pressure on Integration Site Patterns of Lentivirally Transduced Human Hematopoietic Stem Cells." Blood 112, no. 11 (November 16, 2008): 4622. http://dx.doi.org/10.1182/blood.v112.11.4622.4622.

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Abstract Hematologic side effects of cancer chemotherapy like myelosuppression are frequently dose-limiting. Lentiviral gene therapy with cytostatic drug resistance gene transfer to human hematopoietic stem cells (CD34+) is a promising approach to overcome this problem. In this context it is of interest if chemotherapy mediated selection has an impact on lentiviral integration site patterns of transduced hematopoietic stem cells (CD34+). Concerning this issue, human CD34+ cells transduced with a lentiviral self-inactivating (SIN) vector encoding MGMTP140K (the O6-BG resistant mutant of O6-meth

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Persons, Derek A., Phillip W. Hargrove, Esther R. Allay, Hideki Hanawa та Arthur W. Nienhuis. "The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number". Blood 101, № 6 (15 березня 2003): 2175–83. http://dx.doi.org/10.1182/blood-2002-07-2211.

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Increased fetal hemoglobin (HbF) levels diminish the clinical severity of β-thalassemia and sickle cell anemia. A treatment strategy using autologous stem cell–targeted gene transfer of a γ-globin gene may therefore have therapeutic potential. We evaluated oncoretroviral- and lentiviral-based γ-globin vectors for expression in transduced erythroid cell lines. Compared with γ-globin, oncoretroviral vectors containing either a β-spectrin or β-globin promoter and the α-globin HS40 element, a γ-globin lentiviral vector utilizing the β-globin promoter and elements from the β-globin locus control re

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Fichter, Christina, Anupriya Aggarwal, Andrew Kam Ho Wong, Samantha McAllery, Vennila Mathivanan, Bailey Hao, Hugh MacRae, et al. "Modular Lentiviral Vectors for Highly Efficient Transgene Expression in Resting Immune Cells." Viruses 13, no. 6 (June 18, 2021): 1170. http://dx.doi.org/10.3390/v13061170.

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Gene/cell therapies are promising strategies for the many presently incurable diseases. A key step in this process is the efficient delivery of genes and gene-editing enzymes to many cell types that may be resistant to lentiviral vector transduction. Herein we describe tuning of a lentiviral gene therapy platform to focus on genetic modifications of resting CD4+ T cells. The motivation for this was to find solutions for HIV gene therapy efforts. Through selection of the optimal viral envelope and further modification to its expression, lentiviral fusogenic delivery into resting CD4+ T cells ex

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Agosto, Luis M., Jianqing J. Yu, Megan K. Liszewski, Clifford Baytop, Nikolay Korokhov, Laurent M. Humeau, and Una O'Doherty. "The CXCR4-Tropic Human Immunodeficiency Virus Envelope Promotes More-Efficient Gene Delivery to Resting CD4+ T Cells than the Vesicular Stomatitis Virus Glycoprotein G Envelope." Journal of Virology 83, no. 16 (June 3, 2009): 8153–62. http://dx.doi.org/10.1128/jvi.00220-09.

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ABSTRACT Current gene transfer protocols for resting CD4+ T cells include an activation step to enhance transduction efficiency. This step is performed because it is thought that resting cells are resistant to transduction by lentiviral-based gene therapy vectors. However, activating resting cells prior to transduction alters their physiology, with foreseeable and unforeseeable negative consequences. Thus, it would be desirable to transduce resting CD4+ T cells without activation. We recently demonstrated, contrary to the prevailing belief, that wild-type human immunodeficiency virus (HIV) int

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Hargrove, Phillip W., Steven Kepes, Hideki Hanawa, Cheng Cheng, Geoff Neale, Arthur W. Nienhuis, and Derek A. Persons. "Assessment of Changes in Gene Expression Caused by Insertions of a Globin Lentiviral Vector Containing Globin Regulatory Elements or a Lentiviral Vector Containing Retroviral LTR Elements." Blood 104, no. 11 (November 16, 2004): 497. http://dx.doi.org/10.1182/blood.v104.11.497.497.

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Abstract The development of lymphoid leukemia in two children with X-SCID who underwent gene therapy was partially due to activation of the LMO-2 proto-oncogene by the retroviral LTR of the vector which inserted nearby (Hacein-Bey-Abina et al., Science 2003), highlighting the importance of vector design on the potential to activate genes near vector integration sites. As gene therapy vectors for other blood disorders are evaluated, it seems prudent to assess the safety issues regarding insertion for each particular vector in appropriate pre-clinical models. We have focused on developing γ-glob

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D’Costa, Jenice, Heidi M. Brown, Priya Kundra, Alberta Davis-Warren, and Suresh K. Arya. "Human immunodeficiency virus type 2 lentiviral vectors: packaging signal and splice donor in expression and encapsidation." Journal of General Virology 82, no. 2 (February 1, 2001): 425–34. http://dx.doi.org/10.1099/0022-1317-82-2-425.

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Retroviral vectors provide the means for gene transfer with long-term expression. The lentivirus subgroup of retroviruses, such as human immunodeficiency virus type 1 (HIV-1) and type 2 (HIV-2), possesses a number of regulatory and accessory genes and other special elements. These features can be exploited to design vectors for transducing non-dividing as well as dividing cells with the potential for regulated transgene expression. Encapsidation of the transgene RNA in lentiviral vectors is determined by the leader sequence-based multipartite packaging signal. Embedded in the packaging signal

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Sosale, Nisha, Richard K. Tsai, Irena Ivanovska, Philip W. Zoltick, and Dennis E. Discher. "Reducing Immune Response against Lentiviral Vectors: Lentiviral Vector Presentation of CD47, The ‘Marker of Self’." Biophysical Journal 100, no. 3 (February 2011): 403a. http://dx.doi.org/10.1016/j.bpj.2010.12.2393.

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Hanawa, Hideki, Derek A. Persons, and Arthur W. Nienhuis. "Mobilization and Mechanism of Transcription of Integrated Self-Inactivating Lentiviral Vectors." Journal of Virology 79, no. 13 (July 1, 2005): 8410–21. http://dx.doi.org/10.1128/jvi.79.13.8410-8421.2005.

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ABSTRACT Permanent genetic modification of replicating primitive hematopoietic cells by an integrated vector has many potential therapeutic applications. Both oncoretroviral and lentiviral vectors have a predilection for integration into transcriptionally active genes, creating the potential for promoter activation or gene disruption. The use of self-inactivating (SIN) vectors in which a deletion of the enhancer and promoter sequences from the 3′ long terminal repeat (LTR) is copied over into the 5′ LTR during vector integration is designed to improve safety by reducing the risk of mobilizatio

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Herbst, Friederike, Claudia R. Ball, Francesca Tuorto, Wei Wang, Ulrich Kloz, Frank van der Hoeven, Frank Lyko, Christof Von Kalle, and Hanno Glimm. "Impaired Lentiviral Transgene Expression In Vivo Caused by Massive Methylation of SFFV Promoter Sequences." Blood 116, no. 21 (November 19, 2010): 3760. http://dx.doi.org/10.1182/blood.v116.21.3760.3760.

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Abstract Abstract 3760 Lentiviral vectors (LV) assure stable transgene expression in vivo, allowing to investigate genes and their functions. In recent years, lentiviral gene transfer was considered to facilitate the generation of transgenic mice with a higher yield of transgenic offspring as compared to commonly used DNA microinjection. We applied LV to generate a mouse model transgenic for SETBP1 and eGFP. Murine zygotes were infected at dE0.5 with lentiviral particles directly injected into the perivitelline space. Specific PCRs for either the SETBP1 transgene or for the WPRE element of the

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Dijon, Marilyne, Caroline Torne-Celer, Cecile Tonnelle, and Christian Chabannon. "Recombination Occurs in Lentiviral Vectors Designed To Express Both EGFP and EYFP in Human Hematopoietic Cells." Blood 104, no. 11 (November 16, 2004): 5249. http://dx.doi.org/10.1182/blood.v104.11.5249.5249.

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Abstract Recombinant lentiviruses are widely used vectors for in vitro and in vivo long-term gene transfer. Many of the initial gene transfer studies were conducted using single reporter genes. However, for many gene delivery applications, expression and detection of two genes are useful. The goal of this study was to explore the feasibility of directing the independent expression of two marker genes from a single HIV-1 derived lentiviral vector. We used the enhanced green fluorescent protein (EGFP) and the enhanced yellow fluorescent protein (EYFP) as markers. Because of spectral overlap of t

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Mirow, Manuela, Lea Isabell Schwarze, Boris Fehse, and Kristoffer Riecken. "Efficient Pseudotyping of Different Retroviral Vectors Using a Novel, Codon-Optimized Gene for Chimeric GALV Envelope." Viruses 13, no. 8 (July 27, 2021): 1471. http://dx.doi.org/10.3390/v13081471.

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The Gibbon Ape Leukemia Virus envelope protein (GALV-Env) mediates efficient transduction of human cells, particularly primary B and T lymphocytes, and is therefore of great interest in gene therapy. Using internal domains from murine leukemia viruses (MLV), chimeric GALV-Env proteins such as GALV-C4070A were derived, which allow pseudotyping of lentiviral vectors. In order to improve expression efficiency and vector titers, we developed a codon-optimized (co) variant of GALV-C4070A (coGALV-Env). We found that coGALV-Env mediated efficient pseudotyping not only of γ-retroviral and lentiviral v

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Buffa, Viviana, Donatella R. M. Negri, Pasqualina Leone, Roberta Bona, Martina Borghi, Ilaria Bacigalupo, Davide Carlei, Cecilia Sgadari, Barbara Ensoli, and Andrea Cara. "A single administration of lentiviral vectors expressing either full-length human immunodeficiency virus 1 (HIV-1)HXB2 Rev/Env or codon-optimized HIV-1JR-FL gp120 generates durable immune responses in mice." Journal of General Virology 87, no. 6 (June 1, 2006): 1625–34. http://dx.doi.org/10.1099/vir.0.81706-0.

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Genetic immunization using viral vectors provides an effective means to elicit antigen-specific cellular immune responses. Several viral vectors have proven efficacious in inducing immune responses after direct injection in vivo. Among them, recombinant, self-inactivating lentiviral vectors are very attractive delivery systems, as they are able to efficiently transduce into and express foreign genes in a wide variety of mammalian cells. A self-inactivating lentiviral vector was evaluated for the delivery of human immunodeficiency virus 1 (HIV-1) envelope sequences in mice in order to elicit sp

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39

Hanawa, Hideki, Derek A. Persons, Takashi Shimada, and Arthur W. Nienhuis. "Diminished Mobilization of Self-Inactivating (SIN) Lentiviral Vectors Containing Globin Regulatory Elements Compared to Those Containing a Retroviral Long Terminal Repeat." Blood 104, no. 11 (November 16, 2004): 5271. http://dx.doi.org/10.1182/blood.v104.11.5271.5271.

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Abstract Stem cell transfer was successful in treating severe combined immunodeficiency due to deficiencies of the common γ-chain of the lymphoid cytokine receptor (N Engl J Med346:1185, 2002) and adenosine deaminase (Science296:2410, 2002) but 2 of 10 children in the common γ-chain trial developed a lymphoproliferative disease secondary in part due to activation of the LMO2 proto-oncogene by the retroviral long terminal repeat (LTR) (Science302:415, 2003). Both oncoretroviral and lentiviral vectors integrate preferentially into transcriptionally active genes so that vector design to improve s

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Buchschacher, Gary L., and Flossie Wong-Staal. "Development of lentiviral vectors for gene therapy for human diseases." Blood 95, no. 8 (April 15, 2000): 2499–504. http://dx.doi.org/10.1182/blood.v95.8.2499.

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Abstract Retroviral vectors derived from murine retroviruses are being used in several clinical gene therapy trials. Recently, progress has been made in the development of vectors based on the lentivirus genus of retroviruses, which ironically includes a major human pathogen, human immunodeficiency virus (HIV). As these vector systems for clinical gene transfer are developed, it is important to understand the rationale behind their design and development. This article reviews the fundamental features of retrovirus replication and of the elements necessary for development of a retroviral vector

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Buchschacher, Gary L., and Flossie Wong-Staal. "Development of lentiviral vectors for gene therapy for human diseases." Blood 95, no. 8 (April 15, 2000): 2499–504. http://dx.doi.org/10.1182/blood.v95.8.2499.008k35_2499_2504.

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Retroviral vectors derived from murine retroviruses are being used in several clinical gene therapy trials. Recently, progress has been made in the development of vectors based on the lentivirus genus of retroviruses, which ironically includes a major human pathogen, human immunodeficiency virus (HIV). As these vector systems for clinical gene transfer are developed, it is important to understand the rationale behind their design and development. This article reviews the fundamental features of retrovirus replication and of the elements necessary for development of a retroviral vector system,

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42

Herzog, Roland W. "Lentiviral vector for hemophilia gene therapy." Blood 103, no. 10 (May 15, 2004): 3609–10. http://dx.doi.org/10.1182/blood-2004-03-0820.

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Colin, Angélique, Mathilde Faideau, Noelle Dufour, Gwennaelle Auregan, Raymonde Hassig, Thibault Andrieu, Emmanuel Brouillet, Philippe Hantraye, Gilles Bonvento, and Nicole Déglon. "Engineered lentiviral vector targeting astrocytesIn vivo." Glia 57, no. 6 (April 15, 2009): 667–79. http://dx.doi.org/10.1002/glia.20795.

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Stripecke, Renata, Angelo A. Cardoso, Karen A. Pepper, Dianne C. Skelton, Xiao-Jin Yu, Leo Mascarenhas, Kenneth I. Weinberg, Lee M. Nadler, and Donald B. Kohn. "Lentiviral vectors for efficient delivery of CD80 and granulocyte-macrophage– colony-stimulating factor in human acute lymphoblastic leukemia and acute myeloid leukemia cells to induce antileukemic immune responses." Blood 96, no. 4 (August 15, 2000): 1317–26. http://dx.doi.org/10.1182/blood.v96.4.1317.

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Abstract Cell vaccines engineered to express immunomodulators have shown feasibility in eliminating leukemia in murine models. Vectors for efficient gene delivery to primary human leukemia cells are required to translate this approach to clinical trials. In this study, second-generation lentiviral vectors derived from human immunodeficiency virus 1 were evaluated, with the cytomegalovirus (CMV) promoter driving expression of granulocyte-macrophage–colony-stimulating factor (GM-CSF) and CD80 in separate vectors or in a bicistronic vector. The vectors were pseudotyped with vesicular stomatitis v

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45

Stripecke, Renata, Angelo A. Cardoso, Karen A. Pepper, Dianne C. Skelton, Xiao-Jin Yu, Leo Mascarenhas, Kenneth I. Weinberg, Lee M. Nadler, and Donald B. Kohn. "Lentiviral vectors for efficient delivery of CD80 and granulocyte-macrophage– colony-stimulating factor in human acute lymphoblastic leukemia and acute myeloid leukemia cells to induce antileukemic immune responses." Blood 96, no. 4 (August 15, 2000): 1317–26. http://dx.doi.org/10.1182/blood.v96.4.1317.h8001317_1317_1326.

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Cell vaccines engineered to express immunomodulators have shown feasibility in eliminating leukemia in murine models. Vectors for efficient gene delivery to primary human leukemia cells are required to translate this approach to clinical trials. In this study, second-generation lentiviral vectors derived from human immunodeficiency virus 1 were evaluated, with the cytomegalovirus (CMV) promoter driving expression of granulocyte-macrophage–colony-stimulating factor (GM-CSF) and CD80 in separate vectors or in a bicistronic vector. The vectors were pseudotyped with vesicular stomatitis virus G gl

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46

Duvergé, Alexis, and Matteo Negroni. "Pseudotyping Lentiviral Vectors: When the Clothes Make the Virus." Viruses 12, no. 11 (November 16, 2020): 1311. http://dx.doi.org/10.3390/v12111311.

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Delivering transgenes to human cells through transduction with viral vectors constitutes one of the most encouraging approaches in gene therapy. Lentivirus-derived vectors are among the most promising vectors for these approaches. When the genetic modification of the cell must be performed in vivo, efficient specific transduction of the cell targets of the therapy in the absence of off-targeting constitutes the Holy Grail of gene therapy. For viral therapy, this is largely determined by the characteristics of the surface proteins carried by the vector. In this regard, an important property of

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47

Mikkola, Hanna, Niels-Bjarne Woods, Marketa Sjögren, Hildur Helgadottir, Isao Hamaguchi, Sten-Eirik Jacobsen, Didier Trono, and Stefan Karlsson. "Lentivirus Gene Transfer in Murine Hematopoietic Progenitor Cells Is Compromised by a Delay in Proviral Integration and Results in Transduction Mosaicism and Heterogeneous Gene Expression in Progeny Cells." Journal of Virology 74, no. 24 (December 15, 2000): 11911–18. http://dx.doi.org/10.1128/jvi.74.24.11911-11918.2000.

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ABSTRACT Human immunodeficiency virus type 1-based lentivirus vectors containing the green fluorescent protein (GFP) gene were used to transduce murine Lin− c-kit+ Sca1+primitive hematopoietic progenitor cells. Following transduction, the cells were plated into hematopoietic progenitor cell assays in methylcellulose and the colonies were scored for GFP positivity. After incubation for 20 h, lentivirus vectors transduced 27.3% ± 6.7% of the colonies derived from unstimulated target cells, but transduction was more efficient when the cells were supported with stem cell factor (SCF) alone (42.0%

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Lewis, Brian C., Nachimuthu Chinnasamy, Richard A. Morgan, and Harold E. Varmus. "Development of an Avian Leukosis-Sarcoma Virus Subgroup A Pseudotyped Lentiviral Vector." Journal of Virology 75, no. 19 (October 1, 2001): 9339–44. http://dx.doi.org/10.1128/jvi.75.19.9339-9344.2001.

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ABSTRACT We are using avian leukosis-sarcoma virus (ALSV) vectors to generate mouse tumor models in transgenic mice expressing TVA, the receptor for subgroup A ALSV. Like other classical retroviruses, ALSV requires cell division to establish a provirus after infection of host cells. In contrast, lentiviral vectors are capable of integrating their viral DNA into the genomes of nondividing cells. With the intention of initiating tumorigenesis in resting, TVA-positive cells, we have developed a system for the preparation of a human immunodeficiency virus type 1 (HIV-1)-based lentiviral vector, ps

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Saedi-Marghmaleki, Mojtaba, Mohammad-Taghi Moradi, Payam Ghasemi-Dehkordi, Leyla Hashemi, and Ali Karimi. "Evaluation of lentiviral vector-based green fluorescent protein expression in human gastric cancer cell line." Journal of Shahrekord University of Medical Sciences 21, no. 5 (October 30, 2019): 204–9. http://dx.doi.org/10.34172/jsums.2019.36.

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Background and aims : Human immunodeficiency virus type 1 (HIV-1) based-lentivirus vector is one of the most promising viral vectors for gene delivery in different cell lines including gastric cell lines. Therefore, the aim of this study was to produce a lentivirus vector for transduction and expression of green fluorescent protein (GFP) in human gastric cancer cell line, AGS. Materials and Methods: In this piece of work, Escherichia coli HB101 was transformed with plasmids psPAX2, pTD, and pMD2.G, following the purification of which their DNA was extracted along with their quantity and qualit

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Schomber, Tibor, Christian P. Kalberer, Aleksandra Wodnar-Filipowicz, and Radek C. Skoda. "Gene silencing by lentivirus-mediated delivery of siRNA in human CD34+ cells." Blood 103, no. 12 (June 15, 2004): 4511–13. http://dx.doi.org/10.1182/blood-2003-07-2397.

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Abstract To derive an efficient system for gene silencing in human hematopoietic stem cells (HSCs) we modified a lentiviral vector for small interfering RNA (siRNA) delivery. For this purpose, an H1 promoter-driven siRNA expression cassette was introduced into a lentiviral vector, and the p53 mRNA was chosen as a target for siRNA-mediated gene silencing. Using the recombinant lentivirus we infected human cord blood-derived CD34+ cells and obtained a transfection efficiency of up to 50%, as determined by expression of enhanced green fluorescent protein (EGFP). In EGFP-positive long-term culture

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