Robust, reversible gene knockdown using a single lentiviral short hairpin RNA vector

Human Gene Therapy

Volumn 21, Issue 8, 2010, Pages 1005-1017

  Brown, Cheryl Y ^a   Sadlon, Timothy ^a   Gargett, Tessa ^a   Melville, Elizabeth ^b   Zhang, Rui ^c   Drabsch, Yvette ^c   Ling, Michael ^e   Strathdee, Craig A ^d   Gonda, Thomas J ^c   Barry, Simon C ^a,b  

^a CHILD HEALTH RESEARCH INSTITUTE   (Australia)

^b UNIVERSITY OF ADELAIDE   (Australia)

^c UNIVERSITY OF QUEENSLAND   (Australia)

^d AMGEN INC   (United States)

^e INSTITUTE OF MEDICAL AND VETERINARY SCIENCE   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

DOXYCYCLINE; LENTIVIRUS VECTOR; PROTEIN MYB; SHORT HAIRPIN RNA; TRANSCRIPTION FACTOR FOXP3;

ARTICLE; BREAST CANCER; CANCER GROWTH; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; GENETIC TRANSDUCTION; HEMATOPOIESIS; HUMAN; HUMAN CELL; REGULATORY T LYMPHOCYTE; RNA VECTOR;

ANIMALS; DOXYCYCLINE; FORKHEAD TRANSCRIPTION FACTORS; GENE EXPRESSION; GENE KNOCKDOWN TECHNIQUES; GENE TARGETING; GENETIC VECTORS; HEK293 CELLS; HUMANS; LENTIVIRUS; MICE; PROTO-ONCOGENE PROTEINS C-MYB; RNA, SMALL INTERFERING; TRANSFECTION;

ANIMALIA;

EID: 77955500315     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2009.107     Document Type: Article

References (48)

1. Aagaard, L., Amarzguioui, M., Sun, G., Santos, L.C., Ehsani, A., Prydz, H., and Rossi, J.J. (2007). A facile lentiviral vector system for expression of doxycycline-inducible shRNAs: Knockdown of the pre-miRNA processing enzyme Drosha. Mol. Ther. 15, 938-945.
2. An, D.S., Xie, Y., Mao, S.H., Morizono, K., Kung, S.K., and Chen, I.S. (2003). Efficient lentiviral vectors for short hairpin RNA delivery into human cells. Hum. Gene Ther. 14, 1207-1212.
3. Anfossi, G., Gewirtz, A.M., and Calabretta, B. (1989). An oligo-mer complementary to c-myb-encoded mRNA inhibits proliferation of human myeloid leukemia cell lines. Proc. Natl. Acad. Sci. U.S.A. 86, 3379-3383.
4. Barry, S., Brzezinski, M., Yanay, O., Seppen, J.E., and Osborne, W.R. (2005). Sustained elevation of neutrophils in rats induced by lentivirus-mediated G-CSF delivery. J. Gene Med. 7, 1510-1516.
5. Barry, S.C., Harder, B., Brzezinski, M., Flint, L.Y., Seppen, J., and Osborne, W.R. (2001). Lentivirus vectors encoding both central polypurine tract and posttranscriptional regulatory element provide enhanced transduction and transgene expression. Hum. Gene Ther. 12, 1103-1108.
6. Bauer, G., Dao, M.A., Case, S.S., Meyerrose, T., Wirthlin, L., Zhou, P., Wang, X., Herrbrich, P., Arevalo, J., Csik, S., Skelton, D.C., Walker, J., Pepper, K., Kohn, D.B., and Nolta, J.A. (2008). In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol. Ther. 16, 1308-1315.
7. Bennett, C.L., Christie, J., Ramsdell, F., Brunkow, M.E., Ferguson, P.J., Whitesell, L., Kelly, T.E., Saulsbury, F.T., Chance, P.F., and Ochs, H.D. (2001). The immune dysregula-tion, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) is caused by mutations of FOXP3. Nat. Genet. 27, 20-21.
8. Biroccio, A., Benassi, B., D'Agnano, I., D'Angelo, C., Buglioni, S., Mottolese, M., Ricciotti, A., Citro, G., Cosimelli, M., Ramsay, R.G., Calabretta, B., and Zupi, G. (2001). c-Myb and Bcl-x overexpression predicts poor prognosis in colorectal cancer: Clinical and experimental findings. Am. J. Pathol. 158, 1289-1299.
9. \+ regulatory T cells. J. Immunol. Methods 327, 53-62.
10. Brummelkamp, T.R., Bernards, R., and Agami, R. (2002). A system for stable expression of short interfering RNAs in mammalian cells. Science 296, 550-553.
11. Brzezinski, M., Yanay, O., Waldron, L., Barry, S.C., and Osborne, W.R. (2007). G-CSF-lentivirus administration in rats provided sustained elevated neutrophil counts and subsequent EPO-lentivirus administration increased hematocrits. J. Gene Med. 9, 571-578.
12. Chang, X., Gao, J.X., Jiang, Q., Wen, J., Seifers, N., Su, L., Godfrey, V.L., Zuo, T., Zheng, P., and Liu, Y. (2005). The Scurfy mutation of FoxP3 in the thymus stroma leads to defective thymopoiesis. J. Exp. Med. 202, 1141-1151.
13. Chen, C., Ridzon, D.A., Broomer, A.J., Zhou, Z., Lee, D.H., Nguyen, J.T., Barbisin, M., Xu, N.L., Mahuvakar, V.R., Andersen, M.R., Lao, K.Q., Livak, K.J., and Guegler, K.J. (2005). Real-time quantification of microRNAs by stem-loop RT-PCR. Nucleic Acids Res. 33, e179.
14. Drabsch, Y., Hugo, H., Zhang, R., Dowhan, D.H., Miao, Y.R., Gewirtz, A.M., Barry, S.C., Ramsay, R.G., and Gonda, T.J. (2007). Mechanism of and requirement for estrogen-regulated MYB expression in estrogen-receptor-positive breast cancer cells. Proc. Natl. Acad. Sci. U.S.A. 104, 13762-13767.
15. Dull, T., Zufferey, R., Kelly, M., Mandel, R.J., Nguyen, M., Trono, D., and Naldini, L. (1998). A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463-8471.
16. \+ regulatory T cells. Nat. Immunol. 4, 330-336.
17. Gonda, T.J., Sheiness, D.K., and Bishop, J.M. (1982). Transcripts from the cellular homologs of retroviral onco-genes: Distribution among chicken tissues. Mol. Cell. Biol. 2, 617-624.
18. Guerin, M., Sheng, Z.M., Andrieu, N., and Riou, G. (1990). Strong association between c-myb and oestrogen-receptor expression in human breast cancer. Oncogene 5, 131-135. (Pubitemid 20044814)
19. Herold, M.J., van den Brandt, J., Seibler, J., and Reichardt, H.M. (2008). Inducible and reversible gene silencing by stable integration of an shRNA-encoding lentivirus in transgenic rats. Proc. Natl. Acad. Sci. U.S.A. 105, 18507-18512.
20. Hori, S., Nomura, T., and Sakaguchi, S. (2003). Control of regulatory T cell development by the transcription factor Foxp3. Science 299, 1057-1061.
21. Hugo, H., Cures, A., Suraweera, N., Drabsch, Y., Purcell, D., Mantamadiotis, T., Phillips, W., Dobrovic, A., Zupi, G., Gonda, T.J., Iacopetta, B., and Ramsay, R.G. (2006). Mutations in the MYB intron I regulatory sequence increase transcription in colon cancers. Genes Chromosomes Cancer 45, 1143-1154.
22. Li, B., and Greene, M.I. (2008). Special regulatory T-cell review: FOXP3 biochemistry in regulatory T cells: How diverse signals regulate suppression. Immunology 123, 17-19.
23. Liu, B., Paton, J.F., and Kasparov, S. (2008). Viral vectors based on bidirectional cell-specific mammalian promoters and tran-scriptional amplification strategy for use in vitro and in vivo. BMC Biotechnol. 8, 49.
24. May, C., Rivella, S., Callegari, J., Heller, G., Gaensler, K.M., Luzzatto, L., and Sadelain, M. (2000). Therapeutic haemoglobin synthesis in b-thalassaemic mice expressing lentivirus-encoded human b-globin. Nature 406, 82-86.
25. Mucenski, M.L., McLain, K., Kier, A.B., Swerdlow, S.H., Schreiner, C.M., Miller, T.A., Pietryga, D.W., Scott, W.J., Jr., and Potter, S.S. (1991). A functional c-myb gene is required for normal murine fetal hepatic hematopoiesis. Cell 65, 677-689.
26. Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F.H., Verma, I.M., and Trono, D. (1996a). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267.
27. Naldini, L., Blomer, U., Gage, F.H., Trono, D., and Verma, I.M. (1996b). Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. U.S.A. 93, 11382-11388.
28. Pluta, K., Diehl, W., Zhang, X.Y., Kutner, R., Bialkowska, A., and Reiser, J. (2007). Lentiviral vectors encoding tetracycline-dependent repressors and transactivators for reversible knockdown of gene expression: A comparative study. BMC Biotechnol. 7, 41.
29. Ramsay, R.G., and Gonda, T.J. (2008). MYB function in normal and cancer cells. Nature Rev. 8, 523-534.
30. Rasmussen, R. (2001). Rapid cycle real-time PCR: Methods and applications. In: Meuer, S., Wittwer, C., and Nakagawara, K., eds. Quantification on the LightCycler Instrument (Springer Press, Heidelberg, Germany) pp. 21-34.
31. Salmon, P., and Trono, D. (2007). Production and titration of lentiviral vectors. In: Zufferey, R., and Trono, D., eds. Current Protocols in Human Genetics (Wiley Interscience, New York) Chapter 12, Unit 12.10.
32. Salmon, P., Kindler, V., Ducrey, O., Chapuis, B., Zubler, R.H., and Trono, D. (2000). High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 96, 3392-3398.
33. Seibler, J., Kleinridders, A., Kuter-Luks, B., Niehaves, S., Brun-ing, J.C., and Schwenk, F. (2007). Reversible gene knockdown in mice using a tight, inducible shRNA expression system. Nucleic Acids Res. 35, e54.
34. Seidler, B., Schmidt, A., Mayr, U., Nakhai, H., Schmid, R.M., Schneider, G., and Saur, D. (2008). A Cre-loxP-based mouse model for conditional somatic gene expression and knockdown in vivo by using avian retroviral vectors. Proc. Natl. Acad. Sci. U.S.A. 105, 10137-10142.
35. Seppen, J., Barry, S.C., Harder, B., and Osborne, W.R. (2001). Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin. Blood 98, 594-596.
36. Szulc, J., Wiznerowicz, M., Sauvain, M.O., Trono, D., and Ae-bischer, P. (2006). A versatile tool for conditional gene expression and knockdown. Nat. Methods 3, 109-116.
37. Taher, T.E., Tulone, C., Fatah, R., D'Acquisto, F., Gould, D.J., and Mageed, R.A. (2008). Repopulation of B-lymphocytes with restricted gene expression using haematopoietic stem cells engineered with lentiviral vectors. Gene Ther. 15, 998-1006.
38. \+ regulatory T cell: A jack of all trades, master of regulation. Nat. Immunol. 9, 239-244.
39. Tang, F., Hajkova, P., Barton, S., Lao, K., and Surani, M. (2006). MicroRNA expression profiling of single whole embryonic stem cells. Nucleic Acids Res. 34, e9
40. ter Brake, O., and Berkhout, B. (2007). Lentiviral vectors that carry anti-HIV shRNAs: Problems and solutions. J. Gene Med. 9, 743-750
41. ter Brake, O., t Hooft, K., Liu, Y.P., Centlivre, M., von Eije, K.J., and Berkhout, B. (2008). Lentiviral vector design for multiple shRNA expression and durable HIV-1 inhibition. Mol. Ther. 16, 557-564.
42. Thompson, M.A., Flegg, R., Westin, E.H., and Ramsay, R.G. (1997). Microsatellite deletions in the c-myb transcriptional attenuator region associated with over-expression in colon tumour cell lines. Oncogene 14, 1715-1723.
43. Torelli, G., Venturelli, D., Coló, A., Zanni, C., Selleri, L., Moretti, L., Calabretta, B., and Torelli, U. (1987). Expression of c-myb protooncogene and other cell cycle-related genes in normal and neoplastic human colonic mucosa. Cancer Res. 47, 5266-5269.
44. van de Wetering, M., Oving, I., Muncan, V., Pon Fong, M.T., Brantjes, H., van Leenen, D., Holstege, F.C., Brummelkamp, T.R., Agami, R., and Clevers, H. (2003). Specific inhibition of gene expression using a stably integrated, inducible small-interfering-RNA vector. EMBO Rep. 4, 609-615.
45. Ventura, A., Meissner, A., Dillon, C.P., McManus, M., Sharp, P.A., Van Parijs, L., Jaenisch, R., and Jacks, T. (2004). Cre-lox-regulated conditional RNA interference from transgenes. Proc. Natl. Acad. Sci. U.S.A. 101, 10380-10385.
46. Westin, E.H., Gallo, R.C., Arya, S.K., Eva, A., Souza, L.M., Baluda, M.A., Aaronson, S.A., and Wong-Staal, F. (1982). Differential expression of the amv gene in human hemato-poietic cells. Proc. Natl. Acad. Sci. U.S.A. 79, 2194-2198.
47. \+ regulatory T cells. Int. Immunol. 16, 1643-1656.
48. Yanay, O., Barry, S.C., Katen, L.J., Brzezinski, M., Flint, L.Y., Christensen, J., Liggitt, D., Dale, D.C., and Osborne, W.R. (2003). Treatment of canine cyclic neutropenia by lentivirus-mediated G-CSF delivery. Blood 102, 2046-2052.