Efficient transduction of simian cells by HIV-1-based lentiviral vectors that contain mutations in the capsid protein

Molecular Therapy

Volumn 15, Issue 5, 2007, Pages 930-937

  Rits, Maarten A N ^a,b   van Dort, Karel A ^a,b   Münk, Carsten ^c   Meijer, Alexander B ^a   Kootstra, Neeltje A ^a,b  

^a SANQUIN RESEARCH   (Netherlands)

^b UNIVERSITY OF AMSTERDAM   (Netherlands)

^c PAUL EHRLICH INSTITUT   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CAPSID PROTEIN; CYCLOPHILIN A; LENTIVIRUS VECTOR;

AMINO ACID SUBSTITUTION; ANIMAL CELL; ARTICLE; BINDING AFFINITY; BINDING SITE; CONTROLLED STUDY; GENE MUTATION; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS 2; MACACA; NONHUMAN; PERIPHERAL BLOOD MONONUCLEAR CELL; PHENOTYPE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS INFECTION;

ANIMALS; BINDING SITES; CAPSID PROTEINS; CELL LINE; CYCLOPHILIN A; GENETIC VECTORS; HIV-1; HIV-2; HUMANS; LEUKOCYTES, MONONUCLEAR; MACACA; MUTATION; REPRODUCIBILITY OF RESULTS; TRANSDUCTION, GENETIC;

ANIMALIA; CERCOPITHECIDAE; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS 2; MACACA MULATTA; MIRIDAE; SIMIAE;

EID: 34247229559     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.sj.6300091     Document Type: Article

References (47)

1. Hacein-Bey-Abina, S, von Kalle, C, Schmidt, M, Le Deist, F, Wulffraat, N and McIntyre, E et al. (2003). A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348: 255-256.
2. Hacein-Bey-Abina, S, Von Kalle, C, Schmidt, M, McCormack, MP, Wulffraat, N and Leboulch, P et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419.
3. Woods, NB, Bottero, V, Schmidt, M, von Kalle, C and Verma, IM (2006). Gene therapy: Therapeutic gene causing lymphoma. Nature 440: 1123.
4. Hofmann, W, Schubert, D, LaBonte, J, Munson, L, Gibson, S and Scammell, J et al. (1999). Species-specific, postentry barriers to primate immunodeficiency virus infection. J Virol 73: 10020-10028.
5. Besnier, C, Takeuchi, Y and Towers, G (2002). Restriction of lentivirus in monkeys. Proc Natl Acad Sci USA 99: 11920-11925.
6. Cowan, S, Hatziioannou, T, Cunningham, T, Muesing, MA, Gottlinger, HG and Bieniasz, PD (2002). Cellular inhibitors with Fv1-like activity restrict human and simian immunodeficiency virus tropism. Proc Natl Acad Sci USA 99: 11914-11919.
7. Münk, C, Brandt, S M, Lucero, G and Landau, NR (2002). A dominant block to HIV-1 replication at reverse transcription in simian cells. Proc Natl Acad Sci USA 99: 13843-143848.
8. Hatziioannou, T, Cowan, S, Goff, SP, Bieniasz, PD and Towers, GJ (2003). Restriction of multiple divergent retroviruses by Lv1 and Ref1. EMBO J 22: 385-394.
9. Stremlau, M, Owens, C M, Perron, M J, Kiessling, M, Autissier, P and Sodroski, J (2004). The cytoplasmic body component TRIMS alpha restricts HIV-1 infection in Old World monkeys. Nature 427: 848-853.
10. Hatziioannou, T, Perez-Caballero, D, Yang, A, Cowan, S and Bieniasz, P D (2004). Retrovirus resistance factors Ref1 and Lv1 are species-specific variants of TRIM5 alpha. Proc Natl Acad Sci USA 101: 10774-10779.
11. Keckesova, Z, Ylinen, L M and Towers, G J (2004). The human and African green monkey TRIM5 alpha genes encode Ref1 and Lv1 retroviral restriction factor activities. Proc Natl Acad Sci USA 101: 10780-10785.
12. Perron, M J, Stremlau, M, Song, B, Ulm, W, Mulligan, RC and Sodroski, J (2004). TRIM5alpha mediates the postentry block to N-tropic murine leukemia viruses in human cells. Proc Natl Acad Sci USA 101: 11827-11832.
13. Yap, MW, Nisole, S, Lynch, C and Stoye, JP (2004). Trim5α protein restricts both HIV-1 and murine leukemia virus. Proc Natl Acad Sci USA 101: 10786-10791.
14. Ylinen, LM, Keckesova, Z, Wilson, SJ, Ranasinghe, S and Towers, GJ (2005). Differential restriction of human immunodeficiency virus type 2 and simian immunodeficiency virus SIVmac by TRIM5 alpha alleles. J Virol 79: 11580-11587.
15. Luban, J, Bossolt, KL, Franke, EK, Kalpana, GV and Goff, SP (1993). Human immunodeficiency virus type 1 Gag protein binds to cyclophilins A and B. Cell 73: 1067-1078.
16. Franke, EK, Yuan, HE and Luban, J (1994). Specific incorporation of cyclophilin A into HIV-1 virions. Nature 372: 359-362.
17. Braaten, D, Aberham, C, Franke, EK, Yin, L, Phares, W and Luban, J (1996). Cyclosporine A-resistant human immunodeficiency virus type 1 mutants demonstrate that Gag encodes the functional target of cyclophilin A. J Virol 70: 5170-5176.
18. Braaten, D, Franke, EK and Luban, J (1996). Cyclophilin A is required for an early step in the life cycle of human immunodeficiency virus type 1 before the initiation of reverse transcription. J Virol 70 3551-3560.
19. Kootstra, NA, Munk, C, Tonnu, N, Landau, NR and Verma, IM (2003). Abrogation of postentry restriction of HIV-1-based lentiviral vector transduction in simian cells. Proc Natl Acad Sci USA 100: 1298-1303.
20. Towers, GJ, Hatziioannou, T, Cowan, S, Goff, SP, Luban, J and Bieniasz, PD (2003). Cyclophilin A modulates the sensitivity of HIV-1 to host restriction factors. Nat Med 9: 1138-1143.
21. Chatterji, U, Bobardt, MD, Stanfield, R, Ptak RG, Pallansch, LA and Ward, PA et al. (2005). Naturally occurring capsid substitutions render HIV-1 cyclophilin A independent in human cells and TRIM-cyclophilin-resistant in Owl monkey cells. J Biol Chem 280 40293-40300.
22. Gao, F, Bailes, E, Robertson, DL, Chen, Y, Rodenburg, CM and Michael, SF et al. (1999). Origin of HIV-1 in the chimpanzee Pan troglodytes troglodytes. Nature 397: 436-441.
23. Braaten, D, Franke, EK and Luban, J (1996). Cyclophilin A is required for the replication of group M human immunodeficiency virus type 1 (HIV-1) and simian immunodeficiency virus SIV(CPZ)GAB but not group O HIV-1 or other primate immunodeficiency viruses. J Virol 70: 4220-4227.
24. Billich, A, Hammerschmid, F, Peichl, P, Wenger, R, Zenke, G and Quesniaux, V et al. (1995). Mode of action of SDZ NIM 811, a nonimmunosuppressive cyclosporin A analog with activity against human immunodeficiency virus (HIV) type 1: Interference with HIV protein-cyclophilin A interactions. J Virol 69: 2451-2461.
25. Gao, F, Yue, L, White, AT, Pappas, PG, Barchue, J and Hanson, AP et al. (1992). Human infection by genetically diverse SIVSM-related HIV-2 in west Africa. Nature 358: 495-499.
26. Sayah, DM and Luban, J (2004). Selection for loss of Ref1 activity in human cells releases human immunodeficiency virus type 1 from cyclophilin A dependence during infection. J Virol 78: 12066-12070.
27. Sokolskaja, E, Sayah, DM and Luban, J (2004). Target cell cyclophilin A modulates human immunodeficiency virus type 1 infectivity. J Virol 78: 12800-12808.
28. Sokolskaja, E, Berthoux, L and Luban, J (2006). Cyclophilin A and TRIM5alpha independently regulate human immunodeficiency virus type 1 infectivity in human cells. J Virol 80: 2855-2862.
29. Keckesova, Z, Ylinen, LM and Towers, GJ (2006). Cyclophilin A renders human immunodeficiency virus type 1 sensitive to Old World monkey but not human TRIM5 alpha antiviral activity. J Virol 80: 4683-4690.
30. Bosco, DA, Eisenmesser, EZ, Pochapsky, S, Sundquist, WI and Kern, D (2002). Catalysis of cis/trans isomerization in native HIV-1 capsid by human cyclophilin A. Proc Natl Acad Sci USA 99: 5247-5252.
31. Howard, BR, Vajdos, FF, Li, S, Sundquist, WI and Hill, CP (2003). Structural insights into the catalytic mechanism of cyclophilin A. Nat Struct Biol 10: 475-481.
32. Bosco, DA and Kern, D (2004). Catalysis and binding of cyclophillin A with different HIV-1 capsid constructs. Biochemistry 43: 6110-6119.
33. Ikeda, Y, Ylinen, LM, Kahar-Bador, M and Towers, GJ (2004). Influence of gag on human immunodeficiency virus type 1 species-specific tropism. J Virol 78: 11816-11822.
34. Hatziioannou, T, Cowan, S, Von Schwedler, UK, Sundquist, WI and Bieniasz, PD (2004). Species-specific tropism determinants in the human immunodeficiency virus type 1 capsid. J Virol 78: 6005-6012.
35. Owens, CM, Song, B, Perron, MJ, Yang, PC, Stremlau, M and Sodroski, J (2004). Binding and susceptibility to postentry restriction factors in monkey cells are specified by distinct regions of the human immunodeficiency virus type 1 capsid. J Virol 78: 5423-5437.
36. Naldini, L, Blomer, U, Gallay, P, Ory, D, Mulligan, R and Gage, FH et al. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263-267.
37. Dull, T, Zufferey, R, Kelly, M, Mandel, RJ, Nguyen, M and Trono, D et al. (1998). A third-generation lentivirus vector with a conditional packaging system. J Virol 72: 8463-8471.
38. Zufferey, R, Dull, T, Mandel, RJ, Bukovsky, A, Quiroz, D and Naldini, L et al. (1998). Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 72: 9873-9880.
39. Gamble, TR, Vajdos, FF, Yoo, S, Worthylake, DK, Houseweart, M and Sundquist, WI et al. (1996). Crystal structure of human cyclophilin A bound to the amino-terminal domain of HIV-1 capsid. Cell 87: 1285-1294.
40. Yoo, S, Myszka, DG, Yeh, C, McMurray, M, Hill, CP and Sundquist, WI (1997). Molecular recognition in the HIV-1 capsid/cyclophilin A complex. J Mol Biol 269: 780-795.
41. Vajdos, FF, Yoo, S, Houseweart, M, Sundquist, WI and Hill, CP (1997). Crystal structure of cyclophilin A complexed with a binding site peptide from the HIV-1 capsid protein. Protein Sci. 6: 2297-2307.
42. Agarwal, PA (2004). Cis/Trans isomerization in HIV-1 capsid protein catalyzed by cyclophlin A: Insights from computational and theoretical studies. Proteins: Structure, Function, and Bioinformatics 56: 449-463.
43. Bukovsky, AA, Weimann, A, Accola, MA and Gottlinger, HG (1997). Transfer of the HIV-1 cyclophilin-binding site to simian immunodeficiency virus from Macaca mulatta can confer both cyclosporin sensitivity and cyclosporin dependence. Proc Natl Acad Sci USA 94: 10943-10948.
44. Berthoux, L, Sebastian, S, Sokolskaja, E and Luban, J (2005). Cyclophilin A is required for TRIM5 (alpha)-mediated resistance to HIV-1 in Old World monkey cells. Proc Natl Acad Sci USA 102: 14849-14853.
45. Stremlau, M, Song, B, Javanbakht, H, Perron, M and Sodroski, J (2006). Cyclophilin A: An auxiliary but not necessary cofactor for TRIM5 alpha restriction of HIV-1. Virology 351: 112-120.
46. Luban, J (1996). Absconding with the chaperone: Essential cyclophilin-Gag interaction in HIV-1 virions. Cell 87: 1157-1159.
47. Aberham, C, Weber, S and Phares, W (1996). Spontaneous mutations in the human immunodeficiency virus type 1 gag gene that affect viral replication in the presence of cyclosporins. J Virol 70: 3536-3544.