Abstract
Lentiviruses are members of a subgroup of enveloped Retroviridae. Lentivirus-based gene delivery vectors have gained popularity in gene therapy field because of their notable potentials in delivering and integrating transgenes into both mitotic active and inactive cells in vitro and in vivo, a characteristic that overcomes many of the barriers of therapeutic gene therapy application. In the past few years, different lentiviral vector systems have been developed based on primate and nonprimate lentiviruses, including human immunodeficiency virus type 1 (HIV-1), simian immunodeficiency virus (SIV), equine infectious anemia virus (EIAV), and feline immunodeficiency virus (FIV) (see refs. 1–4). However, HIV-1-based lentiviral vector remains the best-studied and well-developed lentiviral vector system that has shown high transduction efficiencies targeting many human and experimental animal cell types, including stem cells (embryonic and adult) (5–10), terminally differentiated somatic cells (i.e., neurons) (7,11,12), muscles (7,13,14), skin (15), liver (13,16,17), islet (18,19), lung epithelium (20,21), retina (22,23), primary T lymphocytes (24,25), and fetal tissues (21,26) (Table 1).
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Verma, I. M. and Somia, N. (1997) Gene therapy—promises, problems and prospects. Nature 389, 239–242.
Amado, R. G. and Chen, I. S. (1999) Lentiviral vectors—the promise of gene therapy within reach? Science 285, 674–676.
Federico, M. (1999) Lentiviruses as gene delivery vectors. Curr. Opin. Biotechnol. 10, 448–453.
Chang, L.-J. and Gay, E. E. (2001) The molecular genetics of lentiviral vectors—current and future perspectives. Curr. Gene Ther. 1, 237–251.
Uchida, N., Sutton, R. E., Friera, A. M., et al. (1998) HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 95, 11,939–11,944.
Miyoshi, H., Smith, K. A., Mosier, D. E., Verma, I. M., and Torbett, B. E. (1999) Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283, 682–686.
Chang, L.-J., Urlacher, V., Iwakuma, T., Cui, Y., and Zucali, J. (1999) Efficacy and safety analyses of a recombinant human immunodeficiency virus type 1 derived vector system. Gene Ther. 6, 715–728.
Hamaguchi, I., Woods, N. B., Panagopoulos, I., et al. (2000) Lentivirus vector gene expression during ES cell-derived hematopoietic development in vitro. J. Virol. 74, 10,778–10,784.
Lois, C., Hong, E. J., Pease, S., Brown, E. J., and Baltimore, D. (2002) Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors. Science 295, 868–872.
Wolfgang, M. J., Eisele, S. G., Browne, M. A., et al. (2001) Rhesus monkey placental transgene expression after lentiviral gene transfer into preimplantation embryos. Proc. Natl. Acad. Sci. USA 98, 10,728–10,732.
Naldini, L., Blomer, U., Gage, F. H., Trono, D., and Verma, I. M. (1996) Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. USA 93, 11,382–11,388.
Blomer, U., Naldini, L., Kafri, T., Trono, D., Verma, I. M., and Gage, F. H. (1997) Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J. Virol. 71, 6641–6649.
Kafri, T., Blomer, U., Peterson, D. A., Gage, F. H., and Verma, I. M. (1997) Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat. Genet. 17, 314–317.
Sakoda, T., Kasahara, N., Hamamori, Y., and Kedes, L. (1999) A high-titer lentiviral production system mediates efficient transduction of differentiated cells including beating cardiac myocytes. J. Mol. Cell Cardiol. 31, 2037–2047.
Baek, S. C., Lin, Q., Robbins, P. B., Fan, H., and Khavari, P. A. (2001) Sustainable systemic delivery via a single injection of lentivirus into human skin tissue. Hum. Gene Ther. 12, 1551–1558.
Pfeifer, A., Kessler, T., Yang, M., et al. (2001) Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging. Mol. Ther. 3, 319–322.
Follenzi, A., Sabatino, G., Lombardo, A., Boccaccio, C., and Naldini, L. (2002) Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum. Gene Ther. 13, 243–260.
Gallichan, W. S., Kafri, T., Krahl, T., Verma, I. M., and Sarvetnick, N. (1998) Lentivirus-mediated transduction of islet grafts with interleukin 4 results in sustained gene expression and protection from insulitis. Hum. Gene Ther. 9, 2717–2726.
Giannoukakis, N., Mi, Z., Gambotto, A., et al. (1999) Infection of intact human islets by a lentiviral vector. Gene Ther. 6, 1545–1551.
Goldman, M. J., Lee, P.-S., Yang, J.-S., and Wilson, J. M. (1997) Lentiviral vectors for gene therapy of cystic fibrosis. Hum. Gene Ther. 8, 2261–2268.
Tarantal, A. F., Lee, C. I., Ekert, J. E., et al. (2001) Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches. Mol. Ther. 4, 614–621.
Miyoshi, H., Takahashi, M., Gage, F. H., and Verma, I. M. (1997) Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc. Natl. Acad. Sci. USA 94, 10,319–10,323.
Bainbridge, J. W., Stephens, C., Parsley, K., et al. (2001) In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium. Gene Ther. 8, 1665–1668.
Chinnasamy, D., Chinnasamy, N., Enriquez, M. J., Otsu, M., Morgan, R. A., and Candotti, F. (2000) Lentiviral-mediated gene transfer into human lymphocytes: role of HIV-1 accessory proteins. Blood 96, 1309–1316.
Costello, E., Munoz, M., Buetti, E., Meylan, P. R., Diggelmann, H., and Thali, M. (2000) Gene transfer into stimulated and unstimulated T lymphocytes by HIV-1-derived lentiviral vectors. Gene Ther. 7, 596–604.
Zahler, M. H., Irani, A., Malhi, H., et al. (2000) The application of a lentiviral vector for gene transfer in fetal human hepatocytes. J. Gene Med. 2, 186–193.
Poznansky, M., Lever, A., Bergeron, L., Haseltine, W., and Sodroski, J. (1991) Gene transfer into human lymphocytes by a defective human immunodeficiency virus type 1 vector. J. Virol. 65, 532–536.
Yu, H., Rabson, A. B., Kaul, M., Ron, Y., and Dougherty, J. P. (1996) Inducible human immunodeficienty virus type 1 packaging cell lines. J. Virol. 70, 4530–4537.
Naldini, L., Blomer, U., Gallay, P., et al. (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263–267.
Dull, T., Zufferey, R., Kelly, M., et al. (1998) A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463–8471.
Cui, Y., Iwakuma, T., and Chang, L. J. (1999) Contributions of viral splice sites and cis-regulatory elements to lentivirus vector function. J. Virol. 73, 6171–6176.
Iwakuma, T., Cui, Y., and Chang, L. J. (1999) Self-inactivating lentiviral vectors with U3 and U5 modifications. Virology 261, 120–132.
Lever, A. M. L. (2000) HIV RNA packaging and lentiviral-based vectors. Adv. Pharmacol. 48, 1–28.
Kafri, T., van Praag, H., Gage, F. H., and Verma, I. M. (2000) Lentiviral vectors: regulated gene expression. Mol. Ther. 1, 516–521.
Zennou, V., Petit, C., Guetard, D., Nerhbass, U., Montagnier, L., and Charneau, P. (2000) HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101, 173–185.
Case, S. S., Price, M. A., Jordan, C. T., et al. (1999) Stable transduction of quiescent CD34(+)CD38(−) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc. Natl. Acad. Sci. USA 96, 2988–2993.
Hawley, T. S., Telford, W. G., and Hawley, R. G. (2001) “Rainbow” reporters for multispectral marking and lineage analysis of hematopoietic stem cells. Stem Cells 19, 118–124.
Zolotukhin, S., Potter, M., Hauswirth, W. W., Guy, J., and Muzyczka, N. (1996) A “humanized” green fluorescent protein cDNA adapted for high-level expression in mammalian cells. J. Virol. 70, 4646–4654.
Cui, Y., Golob, J., Kelleher, E., Ye, Z., Pardoll, D., and Cheng, L. (2002) Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells. Blood 99, 399–408.
MacLaren, D. C., Gambhir, S. S., Satyamurthy, N., et al. (1999) Repetitive, non-invasive imaging of the dopamine D2 receptor as a reporter gene in living animals. Gene Ther. 6, 785–791.
Sun, X., Annala, A. J., Yaghoubi, S. S., et al. (2001) Quantitative imaging of gene induction in living animals. Gene Ther. 8, 1572–1579.
Gambhir, S. S., Barrio, J. R., Phelps, M. E., et al. (1999) Imaging adenoviral-directed reporter gene expression in living animals with positron emission tomography. Proc. Natl. Acad. Sci. USA 96, 2333–2338.
Cavazzana-Calvo, M., Hacein-Bey, S., de Saint Basile, G., et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669–672.
Hanawa, H., Kelly, P. F., Nathwani, A. C., et al. (2002) Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol. Ther. 5, 242–251.
Stitz, J., Buchholz, C. J., Engelstadter, M., et al. (2000) Lentiviral vectors pseudotyped with envelope glycoproteins derived from gibbon ape leukemia virus and murine leukemia virus 10A1. Virology 273, 16–20.
Yu, D., Chen, D., Chiu, C., Razmazma, B., Chow, Y. H., and Pang, S. (2001) Prostate-specific targeting using PSA promoter-based lentiviral vectors. Cancer Gene Ther. 8, 628–635.
Lotti, F., Menguzzato, E., Rossi, C., et al. (2002) Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement. J. Virol. 76, 3996–4007.
Sambrook, J. and Russell, D. W. (2001) Molecular cloning: a laboratory manual, Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY.
Zaiss, A.-K., Son, S., and Chang, L.-J. (2002) RNA 3′-readthrough of oncoretrovirus and lentivirus: implications in vector safety and efficacy. J. Virol. 76, 7209–7219.
Higashikawa, F. and Chang, L.-J. (2001) Kinetic analyses of stability of simple and complex retroviral vectors. Virology 280, 124–131.
Author information
Authors and Affiliations
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2003 Humana Press Inc.
About this protocol
Cite this protocol
Cui, Y., Chang, LJ. (2003). Detection and Selection of Lentiviral Vector-Transduced Cells. In: Federico, M. (eds) Lentivirus Gene Engineering Protocols. Methods in Molecular Biology™, vol 229. Humana Press. https://doi.org/10.1385/1-59259-393-3:69
Download citation
DOI: https://doi.org/10.1385/1-59259-393-3:69
Publisher Name: Humana Press
Print ISBN: 978-1-58829-091-5
Online ISBN: 978-1-59259-393-4
eBook Packages: Springer Protocols