Lentivirus Gene Engineering Protocols pp 233-249 | Cite as
SIV Vectors
- 1 Citations
- 1.1k Downloads
Abstract
The recovery of vectors that are suitable for an in vivo gene delivery has been a recurrent theme in gene therapy research over the last decade. Several challenging hurdles need to be overcome to reach such a goal. First, is a need for methods that allow the preparation of vectors at high titers and in culture systems with potential for large scale-up need to be optimized. Second, the gene transfer vectors should not be recognized by the host immune system in order to avoid inactivation. Upon delivery into gene therapy recipients, vectors should also be able to circumvent the numerous biological barriers that are likely to limit their diffusion and biodistribution in the target organism. They should, therefore, be able to recognize specifically and to penetrate cells of the gene therapy target tissue. Third, they should be able to replicate and to express a transgene in cells that are either not or only slowly proliferating, a predominant situation in vivo. Last, but not least, they should be accepted by both ethical and regulatory authorities. In this respect, the development of vectors derived from viruses that are not pathogenic to human may be preferred.
Keywords
Simian Immunodeficiency Virus Transfer Vector Infectious Titer Vector Stock Packaging GenomeReferences
- 1.Nègre, D., Duisit, G., Mangeot, P.-E., Moullier, P., Darlix, J.-L., and Cosset, F.-L. (2002) Lentiviral vectors derived from simian immunodeficiency virus (SIV), in Current Topics in Microbiology and Immunology, Vol. 261 (Trono, D. ed.), Springer-Verlag, Heidelberg, Germany, pp. 53–74.Google Scholar
- 2.Naldini, L., Blömer, U., Gallay, P., et al. (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263–267.PubMedCrossRefGoogle Scholar
- 3.Nègre, D., Mangeot, P., Duisit, G., et al. (2000) Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells. Gene Ther. 7, 1613–1623.PubMedCrossRefGoogle Scholar
- 4.Clements, J. and Zink, M. (1996) Molecular biology and pathogenesis of animal lentivirus infections. Clin. Microbiol. Rev. 9, 100–117.PubMedGoogle Scholar
- 5.Gardner, M., Endres, M., and Barry, P. (1993) The simian retroviruses SIV and SRV, in The Retroviridae (Levy, J., ed.), Vol. 3, Plenum Press, New York, NY, pp. 133–276.Google Scholar
- 6.Mangeot, P. E., Duperrier, K., Nègre, D., et al. (2002) High levels of transduction of human dendritic cells with optimized SIV vectors. Mol. Ther. 5, 283–290.PubMedCrossRefGoogle Scholar
- 7.Guan, Y., Whitney, J., Diallo, K. and Wainberg, M. (2000) Leader sequences downstream of the primer binding site are important for efficient replication of simian immunodeficiency virus. J. Virol. 74, 8854–8860.PubMedCrossRefGoogle Scholar
- 8.Das, A., Klaver, B., Klasens, B., Wamel, J. V., and Berkhout, B. (1997) A conserved hairpin motif in the R-U5 region of the human immunodeficiency virus type 1 RNA genome is essential for replication. J. Virol. 71, 2346–2356.PubMedGoogle Scholar
- 9.Dorman, N. and Lever, A. (2000) Comparison of viral genomic RNA sorting mechanisms in human immunodeficiency virus type 1 (HIV-1), HIV-2, and moloney murine leukemia virus. J. Virol. 74, 11,413–11,417.PubMedCrossRefGoogle Scholar
- 10.Kaye, J. F. and Lever, A. M. (1998) Nonreciprocal packaging of human immunodeficiency virus type 1 and type 2 RNA: a possible role for the p2 domain of Gag in RNA encapsidation. J. Virol. 72, 5877–5885.PubMedGoogle Scholar
- 11.Ilyinskii, P. and Desrosiers, R. (1998) Identification of a sequence element immediately upstream of the polypurine tract that is essential for replication of simian immunodeficiency virus. EMBO J. 17, 3766–3774.PubMedCrossRefGoogle Scholar
- 12.Giavedoni, L. and Yilma, T. (1996) Construction and characterization of replication-competent simian immunodeficiency virus vectors that express gamma interferon. J. Virol. 70, 2247–2251.PubMedGoogle Scholar
- 13.Ruprecht, R. (1999) Live attenuated AIDS viruses as vaccines: promise or peril? Immunol. Rev. 170, 135–149.PubMedCrossRefGoogle Scholar
- 14.Patience, C., Takeuchi, Y., Cosset, F.-L., and Weiss, R. (1998) Packaging of endogenous retroviral sequences in retroviral vectors produced by murine and human packaging cells. J. Virol. 72, 2671–2676.PubMedGoogle Scholar
- 15.Mangeot, P.-E., Nègre, D., Dubois, B., et al. (2000) Development of minimal lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) and their use for the gene transfer in human dendritic cells. J. Virol. 74, 8307–8315.PubMedCrossRefGoogle Scholar
- 16.Schnell, T., Foley, P., Wirth, M., Munch, J., and Uberla, K. (2000) Development of a self-inactivating, minimal lentivirus vector based on simian immunodeficiency virus. Hum. Gene Ther. 11, 439–447.PubMedCrossRefGoogle Scholar
- 17.Wagner, R., Graf, M., Bieler, K., et al. (2000) Rev-independent expression of synthetic gag-pol genes of human immunodeficiency virus type 1 and simian immunodeficiency virus: implications for the safety of lentiviral vectors. Hum. Gene Ther. 11, 2403–2413.PubMedCrossRefGoogle Scholar
- 18.Nakajima, T., Nakamaru, K., Ido, E., Terao, K., Hayami, M., and Hasegawa, M. (2000) Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system. Hum. Gene Ther. 11, 1863–1874.PubMedCrossRefGoogle Scholar
- 19.Stitz, J., Muhlebach, M. D., Blomer, U., et al. (2001) A novel lentivirus vector derived from apathogenic simian immunodeficiency virus. Virology 291, 191–197.PubMedCrossRefGoogle Scholar
- 20.Schneider, R., Campbell, M., Nasioulas, G., Felber, B. K., and Pavlakis, G. N. (1997) Inactivation of the human immunodeficiency virus type 1 inhibitory elements allows Rev-independent expression of Gag and Gag/protease and particle formation. J. Virol. 71, 4892–4903.PubMedGoogle Scholar
- 21.Belshan, M., Park, G. S., Bilodeau, P., Stoltzfus, C. M., and Carpenter, S. (2000) Binding of equine infectious anemia virus rev to an exon splicing enhancer mediates alternative splicing and nuclear export of viral mRNAs. Mol. Cell Biol. 20, 3550–3557.PubMedCrossRefGoogle Scholar
- 22.Zufferey, R., Donello, J. E., Trono, D., and Hope, T. J. (1999) Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73, 2886–2892.PubMedGoogle Scholar
- 23.Ott, D. E. (1997) Cellular proteins in HIV virions. Rev. Med. Virol. 7, 167–180.PubMedCrossRefGoogle Scholar
- 24.Swanstrom, R. and Wills, J. W. (1997) Synthesis, assembly, and processing of viral proteins, in Retroviruses (Coffin, J. M., Hughes, S. H., and Varmus, H. E., eds.), Cold Spring Harbor Laboratory Press, New York, pp. 263–334.Google Scholar
- 25.Christodoulopoulos, I. and Cannon, P. (2001) Sequences in the cytoplasmic tail of the gibbon ape leukemia virus envelope protein that prevent its incorporation into lentivirus vectors. J. Virol. 75, 4129–4138.PubMedCrossRefGoogle Scholar
- 26.Desmaris, N., Bosch, A., Salaun, C., et al. (2001) Production and neurotropism of lentivirus vectors pseudotyped with lyssavirus envelope glycoproteins. Mol. Ther. 4, 149–156.PubMedCrossRefGoogle Scholar
- 27.Kobinger, G. P., Weiner, D. J., Yu, Q. C., and Wilson, J. M. (2001) Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat. Biotechnol. 19, 225–230.PubMedCrossRefGoogle Scholar
- 28.Lewis, B. C., Chinnasamy, N., Morgan, R. A., and Varmus, H. E. (2001) Development of an avian leukosis-sarcoma virus subgroup A pseudotyped lentiviral vector. J. Virol. 75, 9339–9344.PubMedCrossRefGoogle Scholar
- 29.Mochizuki, H., Schwartz, J. P., Tanaka, K., Brady, R. O., and Reiser, J. (1998) High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J. Virol. 72, 8873–8883.PubMedGoogle Scholar
- 30.Salmon, P., Nègre, D., Trono, D., and Cosset, F.-L. (2000) A chimeric GALV-derived envelope glycoprotein harboring the cytoplasmic tail of MLV envelope efficiently pseudotypes HIV-1 vectors. J. Gen. Med. 2(Suppl.), 23.Google Scholar
- 31.Sandrin, V., Boson, B., Salmon, P., et al. (2002) Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and non-human primates. Blood 100, 823–832.PubMedCrossRefGoogle Scholar
- 32.Stitz, J., Buchholz, C., Engelstadter, M., et al. (2000) Lentiviral vectors pseudo-typed with envelope glycoproteins derived from gibbon ape leukemia virus and murine leukemia virus 10A1. Virology 273, 16–20.PubMedCrossRefGoogle Scholar
- 33.Gatlin, J., Melkus, M. W., Padgett, A., Kelly, P. F., and Garcia, J. V. (2001) Engraftment of NOD/SCID mice with human CD34+ cells transduced by concentrated oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. J. Virol. 75, 9995–9999.PubMedCrossRefGoogle Scholar
- 34.Goerner, M., Horn, P. A., Peterson, L., et al. (2001) Sustained multilineage gene persistence and expression in dogs transplanted with CD34(+) marrow cells transduced by RD114-pseudotype oncoretrovirus vectors. Blood 98, 2065–2070.PubMedCrossRefGoogle Scholar
- 35.Kelly, P., Vandergriff, J., Nathwani, A., Nienhuis, A., and Vanin, E. (2000) Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. Blood 96, 1206–1214.PubMedGoogle Scholar
- 36.Marandin, A., Dubart, A., Pflumio, F., et al. (1998) Retroviral-mediated gene transfer into human CD34+/38-primitive cells capable of reconstituting long-term cultures in vitro and in nonobese diabetic-severe combined immunodeficiency mice in vivo. Hum. Gene Ther. 9, 1497–1511.PubMedCrossRefGoogle Scholar
- 37.Movassagh, M., Desmyter, C., Baillou, C., et al. (1998) High-level gene transfer to cord blood progenitors using gibbon ape leukemia virus pseudotyped retroviral vectors and an improved clinically applicable protocol. Hum. Gene Ther. 9, 225–234.PubMedCrossRefGoogle Scholar
- 38.Porter, C. D., Collins, M. K. L., Tailor, C. S., et al. (1996) Comparison of efficiency of infection of human gene therapy target cells via four different retroviral receptors. Hum. Gene Ther. 7, 913–919.PubMedCrossRefGoogle Scholar
- 39.Peng, K. W., Pham, L., Ye, H., et al. (2001) Organ distribution of gene expression after intravenous infusion of targeted and untargeted lentiviral vectors. Gene Ther. 8, 1456–1463.PubMedCrossRefGoogle Scholar
- 40.Burns, J. C., Friedmann, T., Driever, W., Burrascano, M., and Yee, J. K. (1993) Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc. Natl. Acad. Sci. USA 90, 8033–8037.PubMedCrossRefGoogle Scholar
- 41.Seganti, L., Superti, F., Girmenia, C., Melucci, L., and Orsi, N. (1986) Study of receptors for vesicular stomatitis virus in vertebrate and invertebrate cells. Microbiologica 9, 259–267.PubMedGoogle Scholar
- 42.DePolo, N. J., Reed, J. D., Sheridan, P. L., et al. (2000) VSV-G pseudotyped lentiviral vector particles produced in human cells are inactivated by human serum. Mol. Ther. 2, 218–222.PubMedCrossRefGoogle Scholar
- 43.Cosset, F.-L., Takeuchi, Y., Battini, J., Weiss, R., and Collins, M. (1995) High titer packaging cells producing recombinant retroviruses resistant to human serum. J. Virol. 69, 7430–7436.PubMedGoogle Scholar
- 44.Hatziioannou, T., Delahaye, E., Martin, F., Russell, S. J., and Cosset, F.-L. (1999) Retroviral display of functional binding domains fused to the amino-terminus of influenza haemagglutinin. Hum. Gene Ther. 10, 1533–1544.PubMedCrossRefGoogle Scholar
- 45.Cosset, F.-L., Morling, F. J., Takeuchi, Y., et al. (1995) Retroviral retargeting by envelopes expressing an N-terminal binding domain. J. Virol. 69, 6314–6322.PubMedGoogle Scholar
- 46.Fielding, A., Chapel-Fernandes, S., Chadwick, M., Bullough, F., Cosset, F.-L., and Russell, S. (2000) A hyperfusogenic Gibbon ape leukemia virus envelope glycoprotein: targeting of a cytotoxic gene by ligand display. Hum. Gene Ther. 11, 817–826.PubMedCrossRefGoogle Scholar
- 47.Lavillette, D., Russell, S. J., and Cosset, F.-L. (2001) Retargeting gene delivery by surface-engineered retroviral vector particles. Curr. Opin. Biotechnol. 12, 461–466.PubMedCrossRefGoogle Scholar
- 48.Russell, S. J. and Cosset, F.-L. (1999) Modifying the host range properties of retroviral vectors. J. Gene Med. 1, 300–311.PubMedCrossRefGoogle Scholar
- 49.Kuate, S., Wagner, R., and Uberla, K. (2002) Development and characterization of a minimal inducible packaging cell line for simian immunodeficiency virus-based lentiviral vectors. J. Gene Med. 4, 347–355.PubMedCrossRefGoogle Scholar
- 50.Chackerian, B., Haigwood, N. L., and Overbaugh, J. (1995) Characterization of a CD4-expressing macaque cell line that can detect virus after a single replication cycle and can be infected by diverse simian immunodeficiency virus isolates. Virology 213, 386–394.PubMedCrossRefGoogle Scholar
- 51.Duisit, G., Conrath, H., Saleun, S., et al. (2002) Five recombinant SIV pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat. Mol. Ther. 6, 446–454.PubMedCrossRefGoogle Scholar