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1

Tolstoshev, Paul, and W. French Anderson. "Gene expression using retroviral vectors." Current Opinion in Biotechnology 1, no. 1 (1990): 55–61. http://dx.doi.org/10.1016/0958-1669(90)90010-i.

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2

Blø, Magnus, David R. Micklem, and James B. Lorens. "Enhanced gene expression from retroviral vectors." BMC Biotechnology 8, no. 1 (2008): 19. http://dx.doi.org/10.1186/1472-6750-8-19.

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3

Smith, Michael J., Scott D. Gitlin, Catherine M. Browning, et al. "GLI-2 Modulates Retroviral Gene Expression." Journal of Virology 75, no. 5 (2001): 2301–13. http://dx.doi.org/10.1128/jvi.75.5.2301-2313.2001.

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ABSTRACT GLI proteins are involved in the development of mice, humans, zebrafish, Caenorhabditis elegans, Xenopus, andDrosophila. While these zinc finger-containing proteins bind to TG-rich promoter elements and are known to regulate gene expression in C. elegans and Drosophila, mechanistic understanding of how regulation is mediated through naturally occurring transcriptional promoters is lacking. One isoform of human GLI-2 appears to be identical to a factor previously called Tax helper protein (THP), thus named due to its ability to interact with a TG-rich element in the human T-lymphotropi
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4

Hao, De-Long, Chang-Mei Liu, Wen-Ji Dong, et al. "Knockdown of Human p53 Gene Expression in 293-T Cells by Retroviral Vector-mediated Short Hairpin RNA." Acta Biochimica et Biophysica Sinica 37, no. 11 (2005): 779–83. http://dx.doi.org/10.1111/j.1745-7270.2005.00107.x.

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Abstract RNA interference (RNAi) is an evolutionarily conserved process of gene silencing in multiple organisms, which has become a powerful tool for investigating gene function by reverse genetics. Recently, many groups have reported to use synthesized oligonucleotides or siRNA encoding plasmids to induce RNAi in mammalian cells by transfection, but this is still limited in its application, especially when it is necessary to generate long-term gene silencing in vivo. To circumvent this problem, retrovirus- or lentivirus-delivered RNAi has been developed. Here, we described two retroviral syst
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5

Grabarczyk, Piotr, Piotr J. Wysocki, Katarzyna Gryska, and Andrzej Mackiewicz. "Expression of PiT1 and PiT2 retroviral receptors and transduction efficiency of tumor cells." Acta Biochimica Polonica 49, no. 2 (2002): 333–39. http://dx.doi.org/10.18388/abp.2002_3791.

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Recombinant retroviral vectors are still the most common gene delivery vehicles for gene therapy purposes, especially for construction of genetically modified tumor vaccines (GMTV). However, these vehicles are characterized by relatively low titre and in the case of many tumor cell lines, low transduction efficiency. We constructed bicistronic retroviral vector pseudotypes of amphotropic murine leukemia virus (A-MuLV) and gibbon ape leukemia virus (GaLV), encoding enhanced green fluorescent protein (EGFP) as a rapid and easily detectable reporter gene. Transduction of five different human mela
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6

Agarwal, Manju, Timothy W. Austin, Franck Morel, Jingyi Chen, Ernst Böhnlein, and Ivan Plavec. "Scaffold Attachment Region-Mediated Enhancement of Retroviral Vector Expression in Primary T Cells." Journal of Virology 72, no. 5 (1998): 3720–28. http://dx.doi.org/10.1128/jvi.72.5.3720-3728.1998.

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ABSTRACT We have studied retroviral transgene expression in primary human lymphocytes. Our data demonstrate that transgene expression is high in activated primary CD4+ T cells but significantly decreased in mitotically quiescent cells. Incorporation of a DNA fragment from the scaffold attachment region (SAR) of the human beta interferon gene into the vector improved transgene expression, particularly in quiescent cells. The SAR element functioned in an orientation-dependent manner and enhanced expression of Moloney murine leukemia virus- and murine embryonic stem cell-based vectors. Clonal ana
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7

Richardson, C., M. Ward, S. Podda, and A. Bank. "Mouse fetal liver cells lack functional amphotropic retroviral receptors." Blood 84, no. 2 (1994): 433–39. http://dx.doi.org/10.1182/blood.v84.2.433.bloodjournal842433.

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We have been transducing mouse hematopoietic cells with the human MDR1 (MDR) gene in retroviral vectors to determine the optimal conditions for retroviral gene transfer as a model system for potential human gene therapy. In these studies, we have demonstrated transduction and expression of the human MDR gene using ecotropic and amphotropic MDR- retroviral producer lines. To obtain more mouse hematopoietic cells for detailed study, mouse fetal liver cells (FLC) have been used for MDR transduction and expression, and to reconstitute the ablated marrows of live adult mice. FLC contain hematopoiet
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8

Antoniou, Michael N., Kristian Alsbjerg Skipper, and Omer Anakok. "Optimizing Retroviral Gene Expression for Effective Therapies." Human Gene Therapy 24, no. 4 (2013): 363–74. http://dx.doi.org/10.1089/hum.2013.062.

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9

Yee, J. K., J. C. Moores, D. J. Jolly, J. A. Wolff, J. G. Respess, and T. Friedmann. "Gene expression from transcriptionally disabled retroviral vectors." Proceedings of the National Academy of Sciences 84, no. 15 (1987): 5197–201. http://dx.doi.org/10.1073/pnas.84.15.5197.

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10

Anderson, Claire L., and Gwyn T. Williams. "Apoptosis Gene Hunting Using Retroviral Expression Cloning." Scientific World JOURNAL 1 (2001): 33. http://dx.doi.org/10.1100/tsw.2001.154.

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11

Anderson, Claire L., and Gwyn T. Williams. "APOPTOSIS GENE HUNTING USING RETROVIRAL EXPRESSION CLONING." TheScientificWorldJOURNAL 1, S3 (2001): 33. http://dx.doi.org/10.1100/tsw.2001.23.154.

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12

Israel, DI, and RJ Kaufman. "Retroviral-mediated transfer and amplification of a functional human factor VIII gene." Blood 75, no. 5 (1990): 1074–80. http://dx.doi.org/10.1182/blood.v75.5.1074.1074.

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Abstract Hemophilia A results from a deficiency in factor VII (FVIII), a cofactor in the intrinsic pathway of blood coagulation. As an approach toward genetic therapy of this disease, we constructed a retroviral vector encoding human FVIII and a selectable and amplifiable genetic marker, human adenosine deaminase (Ada). A retrovirus packaging line was transfected with this vector and stable transformants were selected for Ada expression. Isolated transformants produced both FVIII activity in the conditioned medium and retrovirus capable of transferring the Ada selectable marker and FVIII expre
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13

Israel, DI, and RJ Kaufman. "Retroviral-mediated transfer and amplification of a functional human factor VIII gene." Blood 75, no. 5 (1990): 1074–80. http://dx.doi.org/10.1182/blood.v75.5.1074.bloodjournal7551074.

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Hemophilia A results from a deficiency in factor VII (FVIII), a cofactor in the intrinsic pathway of blood coagulation. As an approach toward genetic therapy of this disease, we constructed a retroviral vector encoding human FVIII and a selectable and amplifiable genetic marker, human adenosine deaminase (Ada). A retrovirus packaging line was transfected with this vector and stable transformants were selected for Ada expression. Isolated transformants produced both FVIII activity in the conditioned medium and retrovirus capable of transferring the Ada selectable marker and FVIII expression to
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14

Klymiuk, Nikolai, Mathias Müller, Gottfried Brem та Bernhard Aigner. "Phylogeny, recombination and expression of porcine endogenous retrovirus γ2 nucleotide sequences". Journal of General Virology 87, № 4 (2006): 977–86. http://dx.doi.org/10.1099/vir.0.81552-0.

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Endogenous retroviral sequences in the pig genome represent a potential infectious risk in xenotransplantation. Porcine endogenous retrovirus (PERV) γ sequences described to date have been classified into several families. The known infectious, human-tropic PERVs have been assigned to the PERV γ1 subfamilies A, B and C. High copy numbers and full-length clones have also been observed for an additional family, designated PERV γ2. The aim of this study was to examine the PERV γ2 family by analysis of retroviral pro/pol gene sequences. The proviral load was observed to be similar among various pi
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15

Ismail, Said I., Susan M. Kingsman, Alan J. Kingsman, and Mark Uden. "Split-Intron Retroviral Vectors: Enhanced Expression with Improved Safety." Journal of Virology 74, no. 5 (2000): 2365–71. http://dx.doi.org/10.1128/jvi.74.5.2365-2371.2000.

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ABSTRACT The inclusion of retrovirus-derived introns within retrovirus-based expression vectors leads to a fraction of the resulting transcripts being spliced. Such splicing has been shown to markedly improve expression (W. J. Krall et al., Gene Ther. 3:37–48, 1996). One way to improve upon this still further might involve the use of more efficient introns instead of those from the provirus. Currently, however, incorporation of such introns remains self-defeating since they are removed in the nucleus of the producer cell. In the past, elaborate ways to overcome this problem have included the u
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16

Richardson, C., M. Ward, S. Podda, and A. Bank. "Mouse fetal liver cells lack functional amphotropic retroviral receptors." Blood 84, no. 2 (1994): 433–39. http://dx.doi.org/10.1182/blood.v84.2.433.433.

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Abstract We have been transducing mouse hematopoietic cells with the human MDR1 (MDR) gene in retroviral vectors to determine the optimal conditions for retroviral gene transfer as a model system for potential human gene therapy. In these studies, we have demonstrated transduction and expression of the human MDR gene using ecotropic and amphotropic MDR- retroviral producer lines. To obtain more mouse hematopoietic cells for detailed study, mouse fetal liver cells (FLC) have been used for MDR transduction and expression, and to reconstitute the ablated marrows of live adult mice. FLC contain he
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17

Kim, Seon Hee, Seung Shin Yu, Jong Sang Park, Paul D. Robbins, Chung Sun An, and Sunyoung Kim. "Construction of Retroviral Vectors with Improved Safety, Gene Expression, and Versatility." Journal of Virology 72, no. 2 (1998): 994–1004. http://dx.doi.org/10.1128/jvi.72.2.994-1004.1998.

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ABSTRACT Murine leukemia virus (MLV)-based retroviral vectors are the most frequently used gene delivery vehicles. However, the current vectors are still not fully optimized for gene expression and viral titer, and many genetic and biochemical features of MLV-based vectors are poorly understood. We have previously reported that the retroviral vector MFG, where the gene of interest is expressed as a spliced mRNA, is superior in the level of gene expression with respect to other vectors compared in the study. As one approach to developing improved retroviral vectors, we have systematically perfo
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18

Plavec, I., T. Papayannopoulou, C. Maury, and F. Meyer. "A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells." Blood 81, no. 5 (1993): 1384–92. http://dx.doi.org/10.1182/blood.v81.5.1384.bloodjournal8151384.

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Retroviral-mediated gene transfer of human beta-globin provides a model system for the development of somatic gene therapy for hemoglobinopathies. Previous work has shown that mice receiving a transplant of bone marrow cells infected with a retroviral vector containing the human beta-globin gene can express human beta-globin specifically in erythroid cells; however, the level of expression of the transduced globin gene was low (1% to 2% per gene copy as compared with that of the endogenous mouse beta-globin gene). We report here the construction of a recombinant retrovirus vector encoding a hu
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19

Weidhaas, Joanne Barnes, Elizabeth Lloyd Angelichio, Sabine Fenner, and John M. Coffin. "Relationship between Retroviral DNA Integration and Gene Expression." Journal of Virology 74, no. 18 (2000): 8382–89. http://dx.doi.org/10.1128/jvi.74.18.8382-8389.2000.

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ABSTRACT Although retroviruses can integrate their DNA into a large number of sites in the host genome, factors controlling the specificity of integration remain controversial and poorly understood. To assess the effects of transcriptional activity on integration in vivo, we created quail cell clones containing a construct with a minigene cassette, whose expression is controlled by the papilloma virus E2 protein. From these clones we derived transcriptionally active subclones expressing the wild-type E2 protein and transcriptionally silent subclones expressing a mutant E2 protein that binds it
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20

Plachý, Jiří, Jan Kotáb, Petr Divina, Markéta Reinišová, Filip Šenigl, and Jiří Hejnar. "Proviruses Selected for High and Stable Expression of Transduced Genes Accumulate in Broadly Transcribed Genome Areas." Journal of Virology 84, no. 9 (2010): 4204–11. http://dx.doi.org/10.1128/jvi.02511-09.

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ABSTRACT Retroviruses and retrovirus-derived vectors integrate nonrandomly into the genomes of host cells with specific preferences for transcribed genes, gene-rich regions, and CpG islands. However, the genomic features that influence the transcriptional activities of integrated retroviruses or retroviral vectors are poorly understood. We report here the cloning and characterization of avian sarcoma virus integration sites from chicken tumors. Growing progressively, dependent on high and stable expression of the transduced v-src oncogene, these tumors represent clonal expansions of cells bear
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21

Chambers, C. A., J. Kang, and N. Hozumi. "Long term expression of IL-4 in vivo using retroviral-mediated gene transfer." Journal of Immunology 149, no. 9 (1992): 2899–905. http://dx.doi.org/10.4049/jimmunol.149.9.2899.

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Abstract Th cell subsets regulate immune responses by cell-cell interaction and secretion of cytokines. IL-4 is one of the cytokines secreted by Th cells important for cellular and humoral, particularly IgG1 and IgE responses. To study the role of IL-4 in T cell development and regulation of immune responses in vivo, low IgE-responder C57BL/6 mice were reconstituted with bone marrow cells that had been infected with recombinant retrovirus expressing a high level of IL-4. The reconstituted mice expressed retroviral IL-4 transcripts (9/10) even 8 mo postreconstitution. Physiologically significan
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22

Konetschny, C., G. W. Holzer, and F. G. Falkner. "Retroviral Vectors Produced in the Cytoplasmic Vaccinia Virus System Transduce Intron-Containing Genes." Journal of Virology 76, no. 3 (2002): 1236–43. http://dx.doi.org/10.1128/jvi.76.3.1236-1243.2002.

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ABSTRACT Introns and polyadenylation (pA) sites are known to improve transcript stability and nuclear-cytoplasmic transport and are normally present in efficient gene expression vectors. Standard retroviral vectors, however, do not allow the inclusion of such sequence elements, as mRNA processing at internal splice and pA sites interferes with the production of functional full-length vector genomes. In this report we examined the capability of hybrid vaccinia/retroviral vectors to transduce complex gene cassettes with nuclear RNA processing signals within the retroviral genome. A retroviral ve
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23

Heidmann, Odile, Anthony Béguin, Janio Paternina, et al. "HEMO, an ancestral endogenous retroviral envelope protein shed in the blood of pregnant women and expressed in pluripotent stem cells and tumors." Proceedings of the National Academy of Sciences 114, no. 32 (2017): E6642—E6651. http://dx.doi.org/10.1073/pnas.1702204114.

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Capture of retroviral envelope genes is likely to have played a role in the emergence of placental mammals, with evidence for multiple, reiterated, and independent capture events occurring in mammals, and be responsible for the diversity of present day placental structures. Here, we uncover a full-length endogenous retrovirus envelope protein, dubbed HEMO [human endogenous MER34 (medium-reiteration-frequency-family-34) ORF], with unprecedented characteristics, because it is actively shed in the blood circulation in humans via specific cleavage of the precursor envelope protein upstream of the
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24

Bromberg-White, Jennifer L., Craig P. Webb, Veronique S. Patacsil, Cindy K. Miranti, Bart O. Williams, and Sheri L. Holmen. "Delivery of Short Hairpin RNA Sequences by Using a Replication-Competent Avian Retroviral Vector." Journal of Virology 78, no. 9 (2004): 4914–16. http://dx.doi.org/10.1128/jvi.78.9.4914-4916.2004.

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ABSTRACT While recent studies have demonstrated that retroviral vectors can be used to stably express short hairpin RNA (shRNA) to inhibit gene expression, these studies have utilized replication-defective retroviruses. We describe the creation of a replication-competent, Gateway-compatible retroviral vector capable of expressing shRNA that inhibits the expression of specific genes.
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25

Persons, Derek A., James A. Allay, Esther R. Allay, et al. "Retroviral-Mediated Transfer of the Green Fluorescent Protein Gene Into Murine Hematopoietic Cells Facilitates Scoring and Selection of Transduced Progenitors In Vitro and Identification of Genetically Modified Cells In Vivo." Blood 90, no. 5 (1997): 1777–86. http://dx.doi.org/10.1182/blood.v90.5.1777.

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Abstract We have investigated the utility of the green fluorescent protein (GFP) to serve as a marker to assess retroviral gene transfer into hematopoietic cells and as a tool to identify and enrich for cells expressing high levels of the vector-encoded transcript. GFP, by virtue of a naturally occurring chromophore encoded in its primary sequence, displays autonomous fluorescence, thus eliminating the need for antibody or cytochemical staining to detect its expression. A bicistronic murine stem cell virus (MSCV)-based retroviral vector was constructed containing the GFP cDNA and a mutant, hum
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26

Su, Lishan, Robert Lee, Mark Bonyhadi, et al. "Hematopoietic Stem Cell–Based Gene Therapy for Acquired Immunodeficiency Syndrome: Efficient Transduction and Expression of RevM10 in Myeloid Cells In Vivo and In Vitro." Blood 89, no. 7 (1997): 2283–90. http://dx.doi.org/10.1182/blood.v89.7.2283.

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Abstract Gene delivery via the hematopoietic stem cell (HSC) offers an attractive means to introduce antiviral genes into both T cells and macrophages for acquired immunodeficiency syndrome (AIDS) gene therapy. An amphotropic retroviral vector encoding a bicistronic gene coexpressing RevM10 and the murine CD8α′ chain (lyt2) was developed to transduce HSC/progenitor cells. After transduction of CD34+ cells isolated from human umbilical cord blood, the lyt2 molecule detected by flow cytometry was used to monitor the level of gene transduction and expression and to enrich RevM10-expressing cells
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27

DelaFlor-Weiss, E., C. Richardson, M. Ward, et al. "Transfer and expression of the human multidrug resistance gene in mouse erythroleukemia cells." Blood 80, no. 12 (1992): 3106–11. http://dx.doi.org/10.1182/blood.v80.12.3106.3106.

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Abstract Gene therapy in humans requires the transplantation of genetically modified cells, and it is important to select only those cells capable of expressing high levels of protein from the transferred gene. Expression of the human multiple drug resistance (MDR) gene confers resistance to a variety of compounds in vitro and in vivo. To determine the feasibility of conferring recipient erythroid cells with the MDR phenotype, we have transduced mouse erythroleukemia cells (MELC) with the MDR gene in a retroviral vector. We show here that MELC clones resistant to exposure to colchicine (an MDR
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28

DelaFlor-Weiss, E., C. Richardson, M. Ward, et al. "Transfer and expression of the human multidrug resistance gene in mouse erythroleukemia cells." Blood 80, no. 12 (1992): 3106–11. http://dx.doi.org/10.1182/blood.v80.12.3106.bloodjournal80123106.

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Gene therapy in humans requires the transplantation of genetically modified cells, and it is important to select only those cells capable of expressing high levels of protein from the transferred gene. Expression of the human multiple drug resistance (MDR) gene confers resistance to a variety of compounds in vitro and in vivo. To determine the feasibility of conferring recipient erythroid cells with the MDR phenotype, we have transduced mouse erythroleukemia cells (MELC) with the MDR gene in a retroviral vector. We show here that MELC clones resistant to exposure to colchicine (an MDR-responsi
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29

Yee, J. K., D. J. Jolly, J. C. Moores, J. G. Respess, and T. Friedmann. "Gene Expression from a Transcriptionally Disabled Retroviral Vector." Cold Spring Harbor Symposia on Quantitative Biology 51 (January 1, 1986): 1021–26. http://dx.doi.org/10.1101/sqb.1986.051.01.117.

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30

Saylors, R. L., K. C. Stine, and J. Derrick. "Hematopoietic cytokine-inducible gene expression from retroviral vectors." Gene Therapy 6, no. 5 (1999): 944–46. http://dx.doi.org/10.1038/sj.gt.3300872.

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31

Corey, CA, AD DeSilva, CA Holland, and DA Williams. "Serial transplantation of methotrexate-resistant bone marrow: protection of murine recipients from drug toxicity by progeny of transduced stem cells." Blood 75, no. 2 (1990): 337–43. http://dx.doi.org/10.1182/blood.v75.2.337.337.

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Abstract Recombinant retroviral vectors have been used to transfer a variety of genetic sequences into hematopoietic stem cells. Although transfer and expression of foreign genetic sequences into reconstituting stem cells is one approach to somatic gene therapy, few studies have shown long lasting phenotypic changes in recipient mice in vivo. In this study, we show successful transfer of a methotrexate-resistant cDNA (DHFRr) into reconstituting hematopoietic stem cells using a retroviral vector, FrDHFRr, in which the DHFR cDNA is expressed off a hybrid Friend/Moloney long term repeat. Both pri
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32

Corey, CA, AD DeSilva, CA Holland, and DA Williams. "Serial transplantation of methotrexate-resistant bone marrow: protection of murine recipients from drug toxicity by progeny of transduced stem cells." Blood 75, no. 2 (1990): 337–43. http://dx.doi.org/10.1182/blood.v75.2.337.bloodjournal752337.

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Recombinant retroviral vectors have been used to transfer a variety of genetic sequences into hematopoietic stem cells. Although transfer and expression of foreign genetic sequences into reconstituting stem cells is one approach to somatic gene therapy, few studies have shown long lasting phenotypic changes in recipient mice in vivo. In this study, we show successful transfer of a methotrexate-resistant cDNA (DHFRr) into reconstituting hematopoietic stem cells using a retroviral vector, FrDHFRr, in which the DHFR cDNA is expressed off a hybrid Friend/Moloney long term repeat. Both primary and
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33

Plavec, I., T. Papayannopoulou, C. Maury, and F. Meyer. "A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells." Blood 81, no. 5 (1993): 1384–92. http://dx.doi.org/10.1182/blood.v81.5.1384.1384.

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Abstract Retroviral-mediated gene transfer of human beta-globin provides a model system for the development of somatic gene therapy for hemoglobinopathies. Previous work has shown that mice receiving a transplant of bone marrow cells infected with a retroviral vector containing the human beta-globin gene can express human beta-globin specifically in erythroid cells; however, the level of expression of the transduced globin gene was low (1% to 2% per gene copy as compared with that of the endogenous mouse beta-globin gene). We report here the construction of a recombinant retrovirus vector enco
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34

Zhong, Qiu, Peter Oliver, Weitao Huang, et al. "Efficient c-kit Receptor-Targeted Gene Transfer to Primary Human CD34-Selected Hematopoietic Stem Cells." Journal of Virology 75, no. 21 (2001): 10393–400. http://dx.doi.org/10.1128/jvi.75.21.10393-10400.2001.

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ABSTRACT We have previously reported effective gene transfer with a targeted molecular conjugate adenovirus vector through the c-kit receptor in hematopoietic progenitor cell lines. However, a c-kit-targeted recombinant retroviral vector failed to transduce cells, indicating the existence of significant differences for c-kit target gene transfer between these two viruses. Here we demonstrate that conjugation of an adenovirus to a c-kit-retargeted retrovirus vector enables retroviral transduction. This finding suggests the requirement of endosomalysis for successful c-kit-targeted gene transfer
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35

Gough, Peter J., and Elaine W. Raines. "Gene therapy of apolipoprotein E–deficient mice using a novel macrophage-specific retroviral vector." Blood 101, no. 2 (2003): 485–91. http://dx.doi.org/10.1182/blood-2002-07-2131.

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The use of retroviral gene transfer into hematopoietic stem cells for human gene therapy has been hampered by the absence of retroviral vectors that can generate long-lasting, lineage-specific gene expression. We developed self-inactivating retroviral vectors that incorporate gene-regulatory elements from the macrophage-restricted human CD68 gene. Through the transplantation of transduced murine hematopoietic stem cells (HSCs), we show that a vector incorporating a 342–base pair (bp) fragment of 5′ flanking sequence from the CD68 gene, in addition to the CD68 first intron, was able to direct m
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36

Kuhn, Ulrich, Atsushi Terunuma, Wolfgang Pfutzner, Ruth Ann Foster, and Jonathan C. Vogel. "In Vivo Assessment of Gene Delivery to Keratinocytes by Lentiviral Vectors." Journal of Virology 76, no. 3 (2002): 1496–504. http://dx.doi.org/10.1128/jvi.76.3.1496-1504.2002.

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ABSTRACT For skin gene therapy, introduction of a desired gene into keratinocyte progenitor or stem cells could overcome the problem of achieving persistent gene expression in a significant percentage of keratinocytes. Although keratinocyte stem cells have not yet been completely characterized and purified for gene targeting purposes, lentiviral vectors may be superior to retroviral vectors at gene introduction into these stem cells, which are believed to divide and cycle slowly. Our initial in vitro studies demonstrate that lentiviral vectors are able to efficiently transduce nondividing kera
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37

Hafenrichter, DG, X. Wu, SD Rettinger, SC Kennedy, MW Flye, and KP Ponder. "Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes." Blood 84, no. 10 (1994): 3394–404. http://dx.doi.org/10.1182/blood.v84.10.3394.3394.

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Abstract Hepatic gene therapy could be used to treat a number of inherited blood diseases such as hemophilia or thrombophilia. Although liver-directed retroviral transduction can result in long-term gene expression in vivo, the low level of protein production has limited its clinical application. We reasoned that the insertion of liver-specific promoters into retroviral vectors would increase gene expression in vivo. The 347- bp human alpha 1-antitrypsin (hAAT), the 810-bp murine albumin (mAIb), the 490-bp rat phosphoenolpyruvate carboxykinase (rPECK), and the 596- bp rat liver fatty acid bind
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38

Hafenrichter, DG, X. Wu, SD Rettinger, SC Kennedy, MW Flye, and KP Ponder. "Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes." Blood 84, no. 10 (1994): 3394–404. http://dx.doi.org/10.1182/blood.v84.10.3394.bloodjournal84103394.

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Hepatic gene therapy could be used to treat a number of inherited blood diseases such as hemophilia or thrombophilia. Although liver-directed retroviral transduction can result in long-term gene expression in vivo, the low level of protein production has limited its clinical application. We reasoned that the insertion of liver-specific promoters into retroviral vectors would increase gene expression in vivo. The 347- bp human alpha 1-antitrypsin (hAAT), the 810-bp murine albumin (mAIb), the 490-bp rat phosphoenolpyruvate carboxykinase (rPECK), and the 596- bp rat liver fatty acid binding prote
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39

Lothrop, CD Jr, ZS al-Lebban, GP Niemeyer, et al. "Expression of a foreign gene in cats reconstituted with retroviral vector infected autologous bone marrow." Blood 78, no. 1 (1991): 237–45. http://dx.doi.org/10.1182/blood.v78.1.237.237.

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Abstract A Moloney murine leukemia virus based retroviral vector was used to transfer the bacterial neomycin resistance gene (neoR) into feline hematopoietic cells. We reconstituted four cats that had been lethally irradiated with autologous bone marrow that had been infected with the N2 or SAX retroviral vector. Bone marrow cells from all four cats expressed the neoR gene 30 days posttransplant and three of four cats still had the neoR gene and a low level of drug resistant colony- forming unit granulocyte-macrophage after more than 200 days. Two of the four cats unexpectedly developed diabet
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40

Lothrop, CD Jr, ZS al-Lebban, GP Niemeyer, et al. "Expression of a foreign gene in cats reconstituted with retroviral vector infected autologous bone marrow." Blood 78, no. 1 (1991): 237–45. http://dx.doi.org/10.1182/blood.v78.1.237.bloodjournal781237.

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Abstract:
A Moloney murine leukemia virus based retroviral vector was used to transfer the bacterial neomycin resistance gene (neoR) into feline hematopoietic cells. We reconstituted four cats that had been lethally irradiated with autologous bone marrow that had been infected with the N2 or SAX retroviral vector. Bone marrow cells from all four cats expressed the neoR gene 30 days posttransplant and three of four cats still had the neoR gene and a low level of drug resistant colony- forming unit granulocyte-macrophage after more than 200 days. Two of the four cats unexpectedly developed diabetes mellit
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41

Moore, Kateri A., Frederick A. Fletcher, Raye Lynn Alford, et al. "Expression vectors for human adenosine deaminase gene therapy." Genome 31, no. 2 (1989): 832–39. http://dx.doi.org/10.1139/g89-146.

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Somatic gene transfer offers a possible new approach for treatment of human genetic disease. Defects affecting blood-forming tissues are candidates for therapies involving transfer of genetic information into hematopoietic stem cells. Adenosine deaminase (ADA) deficiency is being used as a model disease for which gene transfer techniques can be developed and evaluated. We describe here the construction and testing of 20 retroviral vectors for their ability to transfer and express human ADA in vitro and in vivo via a mouse bone marrow transplantation model. After infection of primary bone marro
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42

LERNER, NORMA, STEVEN BRIGHAM, STEPHEN GOFF та ARTHUR BANK. "Human β-Globin Gene Expression after Gene Transfer Using Retroviral Vectors". DNA 6, № 6 (1987): 573–82. http://dx.doi.org/10.1089/dna.1987.6.573.

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43

Anderson, Kathryn D., John A. Thompson, Judith M. DiPietro, Kate T. Montgomery, Lola M. Reid, and W. French Anderson. "Gene expression in implanted rat hepatocytes following retroviral-mediated gene transfer." Somatic Cell and Molecular Genetics 15, no. 3 (1989): 215–27. http://dx.doi.org/10.1007/bf01534872.

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44

Eglitis, M., P. Kantoff, E. Gilboa, and W. Anderson. "Gene expression in mice after high efficiency retroviral-mediated gene transfer." Science 230, no. 4732 (1985): 1395–98. http://dx.doi.org/10.1126/science.2999985.

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45

Pollok, Karen E., Helmut Hanenberg, Timothy W. Noblitt, et al. "High-Efficiency Gene Transfer into Normal and Adenosine Deaminase-Deficient T Lymphocytes Is Mediated by Transduction on Recombinant Fibronectin Fragments." Journal of Virology 72, no. 6 (1998): 4882–92. http://dx.doi.org/10.1128/jvi.72.6.4882-4892.1998.

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ABSTRACT Primary human T lymphocytes are powerful targets for genetic modification, although the use of these targets in human gene therapy protocols has been hampered by low levels of transduction. We have shown previously that significant increases in the transduction of hematopoietic stem and progenitor cells with retroviral vectors can be obtained by the colocalization of the retrovirus and target cells on specific fibronectin (FN) adhesion domains (H. Hanenberg, X. L. Xiao, D. Dilloo, K. Hashino, I. Kato, and D. A. Williams, Nat. Med. 2:876–882, 1996). We studied the transfer of genes int
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46

Whitwam, Todd, Mark E. Haskins, Paula S. Henthorn, et al. "Retroviral Marking of Canine Bone Marrow: Long-Term, High-Level Expression of Human Interleukin-2 Receptor Common Gamma Chain in Canine Lymphocytes." Blood 92, no. 5 (1998): 1565–75. http://dx.doi.org/10.1182/blood.v92.5.1565.

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Abstract Optimization of retroviral gene transfer into hematopoietic cells of the dog will facilitate gene therapy of canine X-linked severe combined immunodeficiency (XSCID) and in turn advance similar efforts to treat human XSCID. Both canine and human XSCID are caused by defects in the common γ chain, γc, of receptors for interleukin-2 and other cytokines. In this study, normal dogs were given retrovirally transduced bone marrow cells with and without preharvest mobilization by the canine growth factors granulocyte colony-stimulating factor (G-CSF) and stem cell factor (SCF). Harvey sarcoma
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47

Whitwam, Todd, Mark E. Haskins, Paula S. Henthorn, et al. "Retroviral Marking of Canine Bone Marrow: Long-Term, High-Level Expression of Human Interleukin-2 Receptor Common Gamma Chain in Canine Lymphocytes." Blood 92, no. 5 (1998): 1565–75. http://dx.doi.org/10.1182/blood.v92.5.1565.417k12_1565_1575.

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Abstract:
Optimization of retroviral gene transfer into hematopoietic cells of the dog will facilitate gene therapy of canine X-linked severe combined immunodeficiency (XSCID) and in turn advance similar efforts to treat human XSCID. Both canine and human XSCID are caused by defects in the common γ chain, γc, of receptors for interleukin-2 and other cytokines. In this study, normal dogs were given retrovirally transduced bone marrow cells with and without preharvest mobilization by the canine growth factors granulocyte colony-stimulating factor (G-CSF) and stem cell factor (SCF). Harvey sarcoma virus an
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48

Ren, S., BY Wong, J. Li, XN Luo, PM Wong, and GF Atweh. "Production of genetically stable high-titer retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region." Blood 87, no. 6 (1996): 2518–24. http://dx.doi.org/10.1182/blood.v87.6.2518.bloodjournal8762518.

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The ability to generate stable high-titer vectors that give rise to high levels of expression of transduced globin genes in erythroid cells is a prerequisite for effective retroviral-mediated globin gene therapy. The human beta-globin gene with its immediate flanking sequences does not contain all the regulatory elements necessary for regulated high-level and position-independent expression in erythroid cells. The regulatory element known as the beta-globin locus control region (BetaLCR) can provide a linked Beta-globin gene with these properties. However, addition of BetaLCR sequences to a re
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49

Richardson, C., and A. Bank. "Developmental-stage-specific expression and regulation of an amphotropic retroviral receptor in hematopoietic cells." Molecular and Cellular Biology 16, no. 8 (1996): 4240–47. http://dx.doi.org/10.1128/mcb.16.8.4240.

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Expression of the transmembrane receptor protein Ram-1 may be critical to optimizing retroviral gene transfer. Ram-1 acts as both a sodium-dependent phosphate transporter and a receptor for amphotropic retroviruses. We previously reported detectable Ram-1 in murine hematopoietic fetal liver cells (FLC) despite resistance of these cells to amphotropic retroviral transduction (infection). We document here that Ram-1 expression is completely absent in murine yolk sac cells from days 9.5 through 13.5 of ontogeny and first appears at low levels in midgestational FLC between days 13.5 and 14.5. In a
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50

Delviks, Krista A., and Vinay K. Pathak. "Development of Murine Leukemia Virus-Based Self-Activating Vectors That Efficiently Delete the Selectable Drug Resistance Gene during Reverse Transcription." Journal of Virology 73, no. 10 (1999): 8837–42. http://dx.doi.org/10.1128/jvi.73.10.8837-8842.1999.

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ABSTRACT Expression of the selectable drug resistance gene in retroviral vectors used for gene therapy can lead to a decreased expression of the gene of interest and may induce a host immune response, resulting in a decreased efficiency of gene therapy. In this study, we demonstrate that high-frequency deletion of direct repeats, an inherent property of reverse transcriptases, can be used to efficiently excise the drug resistance gene during reverse transcription. One retroviral vector containing a direct repeat deleted the neomycin resistance expression cassette during a single replication cy
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