Real-time quantitative PCR for the design of lentiviral vector analytical assays

Gene Therapy

Volumn 12, Issue , 2005, Pages S36-S50

  Delenda, C ^a   Gaillard, C ^b  

^a GÉNÉTHON   (France)

^b GenoSafe   (France)

Author keywords

Lentiviral vector; Real time PCR; Standardization

Indexed keywords

COMPLEMENTARY DNA; LENTIVIRUS VECTOR; MESSENGER RNA; NUCLEIC ACID; PLASMID DNA; RNA DIRECTED DNA POLYMERASE; VIRUS RNA;

ANALYTIC METHOD; CONFERENCE PAPER; ENZYME ACTIVITY; GENE CASSETTE; GENE EXPRESSION; GENE TRANSFER; HUMAN IMMUNODEFICIENCY VIRUS 1; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; REAL TIME POLYMERASE CHAIN REACTION; STANDARDIZATION; TARGET CELL; TRANSGENE; VIRUS CAPSID; VIRUS GENOME; VIRUS REPLICATION; VIRUS TITRATION;

ANIMALS; BIOTECHNOLOGY; CELL LINE; DNA PRIMERS; DNA, VIRAL; GENE EXPRESSION; GENETIC VECTORS; HUMANS; LENTIVIRUS; LUMINESCENT PROTEINS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; TRANSDUCTION, GENETIC; TRANSGENES; VIRAL LOAD;

HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRUS; MIRIDAE; RNA VIRUSES;

EID: 27544437703     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302614     Document Type: Article

References (97)

1. Zufferey R et al. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 1997; 15: 871-875.
2. Miyoshi H et al. Transduction of human CD34+ cells that mediate long-term engraftment of NODlSCID mice by HIV vectors. Science 1999; 283: 682-686.
3. Metharom P et al. Novel bovine lentiviral vectors based on Jembrana disease virus. J Gene Med 2000; 2: 176-185.
4. An DS et al. Efficient lentiviral vectors for short hairpin RNA delivery into human cells. Hum Gene Ther 2003; 14: 1207-1212.
5. De A, Lewis XZ, Gambhir SS. Noninvasive imaging of lentiviralmediated reporter gene expression in living mice. Mol Ther 2003; 7: 681-691.
6. Ray P et al. Imaging tri-fusion multimodality reporter gene expression in living subjects. Cancer Res 2004; 64: 1323-1330.
7. D'Costa J et al. HIV-2 derived lentiviral vectors: gene transfer in Parkinson's and Fabry disease models in vitro. J Med Virol 2003; 71: 173-182.
8. Sirin O, Park F. Regulating gene expression using self-inactivating lentiviral vectors containing the mifepristone-inducible system. Gene 2003; 323: 67-77.
9. Tonini T, Claudio PP, Giordano A, Romano G. Determination of functional viral titer by drug-resistance colony assay, expression of green fluorescent protein, and beta-galactoside staining. Methods Mol Biol 2004; 285: 149-153.
10. Grutzkau A et al. Detection of intracellular interleukin-8 in human mast cells: flow cytometry as a guide for immunoelec- tron microscopy. J Histochem Cytochem 1997; 45: 935-945.
11. Ghosh C, Iversen PL. Intracellular delivery strategies for antisense phosphorodiamidate morpholino oligomers. Antisense Nucleic Acid Drug Dev 2000; 10: 263-274.
12. Anson DS, Limberis M. An improved beta-galactosidase reporter gene. J Biotechnol 2004; 108: 17-30.
13. Duisit G et al. Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat. Mol Ther 2002; 6: 446-454.
14. Loewen N et al. Long-term, targeted genetic modification of the aqueous humor outflow tract coupled with noninvasive imaging of gene expression in vivo. Invest Ophthalmol Vis Sci 2004; 45: 3091-3098.
15. Doi K et al. Lentiviral transduction of green fluorescent protein in retinal epithelium: evidence of rejection. Vision Res 2002; 42: 551-558.
16. Roy-Burman P et al. Assay for type C virus in mouse sera based on particulate reverse transcriptase activity. J Virol 1976; 19: 1107-1110.
17. Tonini T, Claudio PP, Giordano A, Romano G. Retroviral and lentiviral vector titration by the analysis of the activity of viral reverse transcriptase. Methods Mol Biol 2004; 285: 155-157.
18. Heneine W et al. Detection of reverse transcriptase by a highly sensitive assay in sera from persons infected with human immunodeficiency virus type 1. J Infect Dis 1995; 171: 1210-1216.
19. Eberle J, Seibl R. A new method for measuring reverse transcriptase activity by ELISA. J Virol Methods 1992; 40: 347-356.
20. Silver J, Maudru T, Fujita K, Repaske R. An RT-PCR assay for the enzyme activity of reverse transcriptase capable of detecting single virions. Nucl Acids Res 1993; 21: 3593-3594.
21. Pyra H, Boni J, Schupbach J. Ultrasensitive retrovirus detection by a reverse transcriptase assay based on product enhancement. Proc Natl Acad Sci USA 1994; 91: 1544-1548.
22. Busso M, Resnick L. Development of an assay that detects transcriptionally competent human immunodeficiency virus type one particles. J Virol Methods 1994; 47: 129-139.
23. Sano K et al. Comparable sensitivities for detection of HIV-1 reverse transcriptase (RT) and other polymerases by RT assays requiring no radioisotopic materials. J Virol Methods 1995; 53: 235-244.
24. Arnold BA, Hepler RW, Keller PM. One-step fluorescent probe product-enhanced reverse transcriptase assay. Biotechniques 1998; 25: 98-106.
25. Lovatt A et al. High throughput detection of retrovirus- associated reverse transcriptase using an improved fluorescent product enhanced reverse transcriptase assay and its comparison to conventional detection methods. J Virol Methods 1999; 82: 185-200.
26. Sears JF, Khan AS. Single-tube fluorescent product-enhanced reverse transcriptase assay with Ampliwax (STF-PERT) for retrovirus quantitation. J Virol Methods 2003; 108: 139-142.
27. Sears JF, Repaske R, Khan AS. Improved Mg2+-based reverse transcriptase assay for detection of primate retroviruses. J Clin Microbiol 1999; 37: 1704-1708.
28. Andre M, Morgeaux S, Fuchs F. Quantitative detection of RT activity by PERT assay: feasibility and limits to a standardized screening assay for human vaccines. Biologicals 2000; 28: 67-80.
29. Schupbach J. Inductionlactivation and detection of occult viral agents present in mammalian cells. Dev Biol (Basel) 2001; 106: 425-437; discussion 465-475.
30. Kuate S, Wagner R, Uberla K. Development and characterization of a minimal inducible packaging cell line for simian immunodeficiency virus-based lentiviral vectors. J Gene Med 2002; 4: 347-355.
31. Martin-Rendon E et al. New methods to titrate EIAV-based lentiviral vectors. Mol Ther 2002; 5: 566-570.
32. Farson D et al. A new-generation stable inducible packaging cell line for lentiviral vectors. Hum Gene Ther 2001; 12: 981-997.
33. Kirkwood TB, Bangham CR. Cycles, chaos, and evolution in virus cultures: a model of defective interfering particles. Proc Natl Acad Sci USA 1994; 91: 8685-8689.
34. Higashikawa F, Chang L. Kinetic analyses of stability of simple and complex retroviral vectors. Virology 2001; 280: 124-131.
35. Murdoch B et al. A rapid screening procedure for the identification of high-titer retrovirus packaging clones. Gene Therapy 1997; 4: 744-749.
36. Onodera M et al. A simple and reliable method for screening retroviral producer clones without selectable markers. Hum Gene Ther 1997; 8: 1189-1194.
37. Forghani B, Hurst JW, Shell GR. Detection of the human immunodeficiency virus genome with a biotinylated DNA probe generated by polymerase chain reaction. Mol Cell Probes 1991; 5: 221-228.
38. Woo JC et al. Investigation of recombinant human insulin-like growth factor type I in thymus regeneration in the acute stage of experimental FIV infection in juvenile cats. AIDS Res Hum Retroviruses 1999; 15: 1377-1388.
39. Watson A et al. Plasma viremia in macaques infected with simian immunodeficiency virus: plasma viral load early in infection predicts survival. J Virol 1997; 71: 284-290.
40. Dyer JR et al. Comparison of NucliSens and Roche Monitor assays for quantitation of levels of human immunodeficiency virus type 1 RNA in plasma. J Clin Microbiol 1999; 37: 447-449.
41. Cao Y et al. Clinical evaluation of branched DNA signal amplification for quantifying HIV type 1 in human plasma. AIDS Res Hum Retroviruses 1995; 11: 353-361.
42. Elbeik T et al. Comparative analysis of HIV-1 viral load assays on subtype quantification: Bayer Versant HIV-1 RNA 3.0 versus Roche Amplicor HIV-1 Monitor version 1.5. J Acquir Immun Defic Syndr 2002; 29: 330-339.
43. Linnen JM et al. Sensitive detection of genetic variants of HIV-1 and HCV with an HIV-1lHCV assay based on transcription- mediated amplification. J Virol Methods 2002; 102: 139-155.
44. Scherr M et al. Quantitative determination of lentiviral vector particle numbers by real-time PCR. Biotechniques 2001; 31: 520, 522, 524, passim.
45. Sastry L et al. Titering lentiviral vectors: comparison of DNA, RNA and marker expression methods. Gene Therapy 2002; 9: 1155-1162.
46. Ikeda Y et al. Gene transduction efficiency in cells of different species by HIV and EIAV vectors. Gene Therapy 2002; 9: 932-938.
47. Lizee G et al. Real-time quantitative reverse transcriptase- polymerase chain reaction as a method for determining lentiviral vector titers and measuring transgene expression. Hum Gene Ther 2003; 14: 497-507.
48. Towers GJ et al. One step screening of retroviral producer clones by real time quantitative PCR. J Gene Med 1999; 1: 352-359.
49. Butler SL, Hansen MS, Bushman FD. A quantitative assay for HIV DNA integration in vivo. Nat Med 2001; 7: 631-634.
50. Charrier S et al. A lentiviral vector encoding the human Wiskott- Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice. Gene Therapy 2005; 12: 597-606.
51. Scherr M et al. Stable RNA interference (RNAi) as an option for anti-bcr-abl therapy. Gene Therapy 2005; 12: 12-21.
52. Zhang B et al. The significance of controlled conditions in lentiviral vector titration and in the use of multiplicity of infection (MOI) for predicting gene transfer events. Genet Vaccines Ther 2004; 2: 6.
53. Sastry L et al. Evaluation of plasmid DNA removal from lentiviral vectors by benzonase treatment. Hum Gene Ther 2004; 15: 221-226.
54. Trono D. Partial reverse transcripts in virions from human immunodeficiency and murine leukemia viruses. J Virol 1992; 66: 4893-4900.
55. Klarmann GJ, Schauber CA, Preston BD. Template-directed pausing of DNA synthesis by HIV-1 reverse transcriptase during polymerization of HIV-1 sequences in vitro. J Biol Chem 1993; 268: 9793-9802.
56. Saenz DT et al. Unintegrated lentivirus DNA persistence and accessibility to expression in nondividing cells: analysis with class I integrase mutants. J Virol 2004; 78: 2906-2920.
57. Van Maele B, De Rijck J, De Clercq E, Debyser Z. Impact of the central polypurine tract on the kinetics of human immuno- S49 deficiency virus type 1 vector transduction. J Virol 2003; 77: 4685-4694.
58. Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector (see comments). Science 1996; 272: 263-267.
59. Gallichan WS et al. Lentivirus-mediated transduction of islet grafts with interleukin 4 results in sustained gene expression and protection from insulitis. Hum Gene Ther 1998; 9: 2717-2726.
60. Kafri T et al. A packaging cell line for lentivirus vectors. J Virol 1999; 73: 576-584.
61. Dull T et al. A third-generation lentivirus vector with a conditional packaging system. J Virol 1998; 72: 8463-8471.
62. Gasmi M et al. Requirements for efficient production and transduction of human immunodeficiency virus type 1-based vectors. J Virol 1999; 73: 1828-1834.
63. Mochizuki H et al. High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J Virol 1998; 72: 8873-8883.
64. Segall HI, Yoo E, Sutton RE. Characterization and detection of artificial replication-competent lentivirus of altered host range. Mol Ther 2003; 8: 118-129.
65. Sastry L et al. Certification assays for HIV-1-based vectors: frequent passage of gag sequences without evidence of replication-competent viruses. Mol Ther 2003; 8: 830-839.
66. Waddington SN et al. Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice. Gene Therapy 2003; 10: 1234-1240.
67. Srinivasakumar N, Schuening FG. A lentivirus packaging system based on alternative RNA transport mechanisms to express helper and gene transfer vector RNAs and its use to study the requirement of accessory proteins for particle formation and gene delivery. J Virol 1999; 73: 9589-9598.
68. Wu X et al. Development of a novel trans-lentiviral vector that affords predictable safety. Mol Ther 2000; 2: 47-55.
69. Mautino MR, Ramsey WJ, Reiser J, Morgan RA. Modified human immunodeficiency virus-based lentiviral vectors display decreased sensitivity to trans-dominant Rev. Hum Gene Ther 2000; 11: 895-908.
70. Escarpe P et al. Development of a sensitive assay for detection of replication-competent recombinant lentivirus in large-scale HIV-based vector preparations. Mol Ther 2003; 8: 332-341.
71. Administration FaD. Guidance for industry: guidance for human somatic cell therapy and gene therapy http:llwww.fda.govlcberlgdlnslsomgene.pdf. 1991.
72. Pilaro AM, Serabian MA. Preclinical development strategies for novel gene therapeutic products. Toxicol Pathol 1999; 27: 4-7.
73. Administration FaD. Points to consider in human somatic cell therapy and gene therapy http:llwww.fda.govlcberlgdlnsl ptcsomat.pdf1991.
74. Gonin P, Gaillard C. Gene transfer vector biodistribution: pivotal safety studies in clinical gene therapy development. Gene Therapy 2004; 11 (Suppl 1): S98-S108.
75. Kafri T et al. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 1997; 17: 314-317.
76. Johnson LG, Olsen JC, Naldini L, Boucher RC. Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo. Gene Therapy 2000; 7: 568-574.
77. Miyoshi H et al. Development of a self-inactivating lentivirus vector. J Virol 1998; 72: 8150-8157.
78. Woods NB et al. Lentiviral gene transfer into primary and secondary NODlSCID repopulating cells (in process citation). Blood 2000; 96: 3725-3733.
79. Ohashi K, Park F, Kay MA. Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo. Hum Gene Ther 2002; 13: 653-663.
80. Peng KW et al. Organ distribution of gene expression after intravenous infusion of targeted and untargeted lentiviral vectors. Gene Therapy 2001; 8: 1456-1463.
81. VandenDriessche T et al. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen- presenting cells in vivo. Blood 2002; 100: 813-822.
82. Vargas Jr J et al. Novel integrase-defective lentiviral episomal vectors for gene transfer. Hum Gene Ther 2004; 15: 361-372.
83. Pan D et al. Biodistribution and toxicity studies of VSVG- pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow. Mol Ther 2002; 6: 19-29.
84. MacKenzie TC et al. Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol Ther 2002; 6: 349-358.
85. Fleury S et al. Multiply attenuated, self-inactivating lentiviral vectors efficiently deliver and express genes for extended periods of time in adult rat cardiomyocytes in vivo. Circulation 2003; 107: 2375-2382. Epub 2003 Apr 2314.
86. Yamada K et al. Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector. Mol Ther 2003; 8: 600-610.
87. Kurre P et al. Efficient marking of murine long-term repopulating stem cells targeting unseparated marrow cells at low lentiviral vector particle concentration. Mol Ther 2004; 9: 914-922.
88. Hanawa H et al. Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system. Blood 2004; 103: 4062-4069. Epub 2004 Feb 4019.
89. Jimenez DF et al. HIV-1-derived lentiviral vectors and fetal route of administration on transgene biodistribution and expression in rhesus monkeys. Gene Therapy 2005; 7: 7.
90. Morizono K et al. Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection. Nat Med 2005; 11: 346-352. Epub 2005 Feb 2013.
91. Lamikanra A et al. In vivo evaluation of an EIAV vector for the systemic genetic delivery of therapeutic antibodies. Gene Therapy 2005; 17: 17.
92. Di Domenico C et al. Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. Hum Gene Ther 2005; 16: 81-90.
93. Di Natale P et al. Treatment of the mouse model of mucopoly- saccharideosis type IIIB with lentiviral-naglu vector. Biochem J 2005; 13: 13.
94. Indraccolo S et al. Gene transfer in ovarian cancer cells: a comparison between retroviral and lentiviral vectors. Cancer Res 2002; 62: 6099-6107.
95. Woychik RP et al. An inherited limb deformity created by insertional mutagenesis in a transgenic mouse. Nature 1985; 318: 36-40.
96. McNeish JD, Scott Jr WJ, Potter SS. Legless, a novel mutation found in PHT1-1 transgenic mice. Science 1988; 241: 837-839.
97. Follenzi A et al. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet 2000; 25: 217-222.