MicroRNA-like antivirals

Biochimica et Biophysica Acta - Gene Regulatory Mechanisms

Volumn 1809, Issue 11-12, 2011, Pages 746-755

  Arbuthnot, Patrick ^a  

^a UNIVERSITY OF THE WITWATERSRAND   (South Africa)

Author keywords

Dengue virus; HBV; HCV; HIV 1; MicroRNA; RNAi

Indexed keywords

ADENOVIRUS VECTOR; CD34 ANTIGEN; DNA DIRECTED DNA POLYMERASE GAMMA; LENTIVIRUS VECTOR; MICRORNA; PARVOVIRUS VECTOR; SHORT HAIRPIN RNA; SMALL INTERFERING RNA;

ANTIVIRAL ACTIVITY; DENGUE VIRUS; DNA TEMPLATE; GENE CASSETTE; GENE EXPRESSION REGULATION; GENE SILENCING; GENETIC ENGINEERING; GENETIC TRANSDUCTION; HEPATITIS B VIRUS; HEPATITIS C VIRUS; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; REVIEW; RNA INTERFERENCE; RNA SEQUENCE; VIRAL GENE DELIVERY SYSTEM; VIRUS INFECTION; VIRUS RECOMBINANT; VIRUS REPLICATION;

ANIMALS; DENGUE; GENE SILENCING; HEPATITIS B; HEPATITIS C; HIV INFECTIONS; HUMANS; MICRORNAS; RNA INTERFERENCE;

ADENO-ASSOCIATED VIRUS; DENGUE VIRUS; DENGUE VIRUS GROUP; HEPATITIS B VIRUS; HEPATITIS C VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1;

EID: 80655149138     PISSN: 18749399     EISSN: 18764320     Source Type: Journal    
DOI: 10.1016/j.bbagrm.2011.05.006     Document Type: Review

References (134)

1. Elbashir S.M., Harborth J., Lendeckel W., Yalcin A., Weber K., Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411:494-498.
2. Kim V.N., Han J., Siomi M.C. Biogenesis of small RNAs in animals. Nat. Rev. Mol. Cell Biol. 2009, 10:126-139.
3. Arbuthnot P. Harnessing RNA interference for the treatment of viral infections. Drug News Perspect. 2010, 23:341-350.
4. Mourelatos Z., Dostie J., Paushkin S., Sharma A., Charroux B., Abel L., Rappsilber J., Mann M., Dreyfuss G. miRNPs: a novel class of ribonucleoproteins containing numerous microRNAs. Genes Dev. 2002, 16:720-728.
5. Lagos-Quintana M., Rauhut R., Lendeckel W., Tuschl T. Identification of novel genes coding for small expressed RNAs. Science 2001, 294:853-858.
6. Lau N.C., Lim L.P., Weinstein E.G., Bartel D.P. An abundant class of tiny RNAs with probable regulatory roles in Caenorhabditis elegans. Science 2001, 294:858-862.
7. Stefani G., Slack F.J. Small non-coding RNAs in animal development. Nat. Rev. Mol. Cell Biol. 2008, 9:219-230.
8. Lee Y., Ahn C., Han J., Choi H., Kim J., Yim J., Lee J., Provost P., Radmark O., Kim S., Kim V.N. The nuclear RNase III Drosha initiates microRNA processing. Nature 2003, 425:415-419.
9. Lee Y., Kim M., Han J., Yeom K.H., Lee S., Baek S.H., Kim V.N. MicroRNA genes are transcribed by RNA polymerase II. EMBO J. 2004, 23:4051-4060.
10. Bogerd H.P., Karnowski H.W., Cai X., Shin J., Pohlers M., Cullen B.R. A mammalian herpesvirus uses noncanonical expression and processing mechanisms to generate viral MicroRNAs. Mol. Cell 2010, 37:135-142.
11. Berezikov E., Chung W.J., Willis J., Cuppen E., Lai E.C. Mammalian mirtron genes. Mol. Cell 2007, 28:328-336.
12. Ruby J.G., Jan C.H., Bartel D.P. Intronic microRNA precursors that bypass Drosha processing. Nature 2007, 448:83-86.
13. Babiarz J.E., Ruby J.G., Wang Y., Bartel D.P., Blelloch R. Mouse ES cells express endogenous shRNAs, siRNAs, and other microprocessor-independent, Dicer-dependent small RNAs. Genes Dev. 2008, 22:2773-2785.
14. Yi R., Qin Y., Macara I.G., Cullen B.R. Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs. Genes Dev. 2003, 17:3011-3016.
15. Zhang H., Kolb F.A., Jaskiewicz L., Westhof E., Filipowicz W. Single processing center models for human Dicer and bacterial RNase III. Cell 2004, 118:57-68.
16. Chendrimada T.P., Gregory R.I., Kumaraswamy E., Norman J., Cooch N., Nishikura K., Shiekhattar R. TRBP recruits the Dicer complex to Ago2 for microRNA processing and gene silencing. Nature 2005, 436:740-744.
17. Khvorova A., Reynolds A., Jayasena S.D. Functional siRNAs and miRNAs exhibit strand bias. Cell 2003, 115:209-216.
18. Lewis B.P., Burge C.B., Bartel D.P. Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets. Cell 2005, 120:15-20.
19. Zeng Y., Yi R., Cullen B.R. MicroRNAs and small interfering RNAs can inhibit mRNA expression by similar mechanisms. Proc. Natl. Acad. Sci. U.S.A. 2003, 100:9779-9784.
20. Meister G., Landthaler M., Patkaniowska A., Dorsett Y., Teng G., Tuschl T. Human Argonaute2 mediates RNA cleavage targeted by miRNAs and siRNAs. Mol. Cell 2004, 15:185-197.
21. Cheloufi S., Dos Santos C.O., Chong M.M., Hannon G.J. A dicer-independent miRNA biogenesis pathway that requires Ago catalysis. Nature 2010, 465:584-589.
22. Li L., Lin X., Khvorova A., Fesik S.W., Shen Y. Defining the optimal parameters for hairpin-based knockdown constructs. RNA 2007, 13:1765-1774.
23. Dyer V., Ely A., Bloom K., Weinberg M., Arbuthnot P. tRNA Lys3 promoter cassettes that efficiently express RNAi-activating antihepatitis B virus short hairpin RNAs. Biochem. Biophys. Res. Commun. 2010, 398:640-646.
24. Scherer L.J., Frank R., Rossi J.J. Optimization and characterization of tRNA-shRNA expression constructs. Nucleic Acids Res. 2007, 35:2620-2628.
25. Boden D., Pusch O., Lee F., Tucker L., Ramratnam B. Human immunodeficiency virus type 1 escape from RNA interference. J. Virol. 2003, 77:11531-11535.
26. Das A.T., Brummelkamp T.R., Westerhout E.M., Vink M., Madiredjo M., Bernards R., Berkhout B. Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition. J. Virol. 2004, 78:2601-2605.
27. Westerhout E.M., Ooms M., Vink M., Das A.T., Berkhout B. HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome. Nucleic Acids Res. 2005, 33:796-804.
28. Wilson J.A., Richardson C.D. Hepatitis C virus replicons escape RNA interference induced by a short interfering RNA directed against the NS5b coding region. J. Virol. 2005, 79:7050-7058.
29. Czauderna F., Santel A., Hinz M., Fechtner M., Durieux B., Fisch G., Leenders F., Arnold W., Giese K., Klippel A., Kaufmann J. Inducible shRNA expression for application in a prostate cancer mouse model. Nucleic Acids Res. 2003, 31:e127.
30. DeVincenzo J., Cehelsky J.E., Alvarez R., Elbashir S., Harborth J., Toudjarska I., Nechev L., Murugaiah V., Van Vliet A., Vaishnaw A.K., Meyers R. Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV). Antiviral Res. 2008, 77:225-231.
31. Geisbert T.W., Lee A.C., Robbins M., Geisbert J.B., Honko A.N., Sood V., Johnson J.C., de Jong S., Tavakoli I., Judge A., Hensley L.E., Maclachlan I. Postexposure protection of non-human primates against a lethal Ebola virus challenge with RNA interference: a proof-of-concept study. Lancet 2010, 375:1896-1905.
32. Berkhout B. Toward a durable anti-HIV gene therapy based on RNA interference. Ann. N. Y. Acad. Sci. 2009, 1175:3-14.
33. Berkhout B., ter Brake O. Towards a durable RNAi gene therapy for HIV-AIDS. Expert Opin. Biol. Ther. 2009, 9:161-170.
34. Barichievy S., Saayman S., Arbuthnot P., Weinberg M.S. RNA interference-based gene expression strategies aimed at sustained therapeutic inhibition of HIV. Curr. Top. Med. Chem. 2009, 9:1065-1078.
35. Ratner L., Haseltine W., Patarca R., Livak K.J., Starcich B., Josephs S.F., Doran E.R., Rafalski J.A., Whitehorn E.A., Baumeister K., et al. Complete nucleotide sequence of the AIDS virus, HTLV-III. Nature 1985, 313:277-284.
36. Sanchez-Pescador R., Power M.D., Barr P.J., Steimer K.S., Stempien M.M., Brown-Shimer S.L., Gee W.W., Renard A., Randolph A., Levy J.A., et al. Nucleotide sequence and expression of an AIDS-associated retrovirus (ARV-2). Science 1985, 227:484-492.
37. Wain-Hobson S., Sonigo P., Danos O., Cole S., Alizon M. Nucleotide sequence of the AIDS virus, LAV. Cell 1985, 40:9-17.
38. Boden D., Pusch O., Lee F., Tucker L., Shank P.R., Ramratnam B. Promoter choice affects the potency of HIV-1 specific RNA interference. Nucleic Acids Res. 2003, 31:5033-5038.
39. Ter brake O., Hooft K.t., Liu Y.P., Centlivre M., von Eije K.J., Berkhout B. Lentiviral vector design for multiple shRNA expression and durable HIV-1 inhibition. Mol. Ther. 2008, 16:557-564.
40. Barichievy S., Saayman S., von Eije K.J., Morris K.V., Arbuthnot P., Weinberg M.S. The inhibitory efficacy of RNA POL III-expressed long hairpin RNAs targeted to untranslated regions of the HIV-1 5' long terminal repeat. Oligonucleotides 2007, 17:419-431.
41. Konstantinova P., ter Brake O., Haasnoot J., de Haan P., Berkhout B. Trans-inhibition of HIV-1 by a long hairpin RNA expressed within the viral genome. Retrovirology 2007, 4:15.
42. Liu Y.P., Haasnoot J., Berkhout B. Design of extended short hairpin RNAs for HIV-1 inhibition. Nucleic Acids Res. 2007, 35:5683-5693.
43. Nishitsuji H., Kohara M., Kannagi M., Masuda T. Effective suppression of human immunodeficiency virus type 1 through a combination of short- or long-hairpin RNAs targeting essential sequences for retroviral integration. J. Virol. 2006, 80:7658-7666.
44. Saayman S., Barichievy S., Capovilla A., Morris K.V., Arbuthnot P., Weinberg M.S. The efficacy of generating three independent anti-HIV-1 siRNAs from a single U6 RNA Pol III-expressed long hairpin RNA. PLoS One 2008, 3:e2602.
45. Liu Y.P., von Eije K.J., Schopman N.C., Westerink J.T., ter Brake O., Haasnoot J., Berkhout B. Combinatorial RNAi against HIV-1 using extended short hairpin RNAs. Mol. Ther. 2009, 17:1712-1723.
46. Saayman S., Arbuthnot P., Weinberg M.S. Deriving four functional anti-HIV siRNAs from a single Pol III-generated transcript comprising two adjacent long hairpin RNA precursors. Nucleic Acids Res. 2010, 38:6652-6663.
47. ter Brake O., Konstantinova P., Ceylan M., Berkhout B. Silencing of HIV-1 with RNA interference: a multiple shRNA approach. Mol. Ther. 2006, 14:883-892.
48. Unwalla H.J., Li H.T., Bahner I., Li M.J., Kohn D., Rossi J.J. Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein. J. Virol. 2006, 80:1863-1873.
49. Liu Y.P., Gruber J., Haasnoot J., Konstantinova P., Berkhout B. RNAi-mediated inhibition of HIV-1 by targeting partially complementary viral sequences. Nucleic Acids Res. 2009, 37:6194-6204.
50. Aagaard L.A., Zhang J., von Eije K.J., Li H., Saetrom P., Amarzguioui M., Rossi J.J. Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs. Gene Ther. 2008, 15:1536-1549.
51. Brass A.L., Dykxhoorn D.M., Benita Y., Yan N., Engelman A., Xavier R.J., Lieberman J., Elledge S.J. Identification of host proteins required for HIV infection through a functional genomic screen. Science 2008, 319:921-926.
52. Bushman F.D., Malani N., Fernandes J., D'Orso I., Cagney G., Diamond T.L., Zhou H., Hazuda D.J., Espeseth A.S., Konig R., Bandyopadhyay S., Ideker T., Goff S.P., Krogan N.J., Frankel A.D., Young J.A., Chanda S.K. Host cell factors in HIV replication: meta-analysis of genome-wide studies. PLoS Pathog. 2009, 5:e1000437.
53. Konig R., Zhou Y., Elleder D., Diamond T.L., Bonamy G.M., Irelan J.T., Chiang C.Y., Tu B.P., De Jesus P.D., Lilley C.E., Seidel S., Opaluch A.M., Caldwell J.S., Weitzman M.D., Kuhen K.L., Bandyopadhyay S., Ideker T., Orth A.P., Miraglia L.J., Bushman F.D., Young J.A., Chanda S.K. Global analysis of host-pathogen interactions that regulate early-stage HIV-1 replication. Cell 2008, 135:49-60.
54. Hutter G., Nowak D., Mossner M., Ganepola S., Mussig A., Allers K., Schneider T., Hofmann J., Kucherer C., Blau O., Blau I.W., Hofmann W.K., Thiel E. Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N. Engl. J. Med. 2009, 360:692-698.
55. Anderson J., Akkina R. HIV-1 resistance conferred by siRNA cosuppression of CXCR4 and CCR5 coreceptors by a bispecific lentiviral vector. AIDS Res. Ther. 2005, 2:1.
56. Surabhi R.M., Gaynor R.B. RNA interference directed against viral and cellular targets inhibits human immunodeficiency Virus Type 1 replication. J. Virol. 2002, 76:12963-12973.
57. Ping Y.H., Chu C.Y., Cao H., Jacque J.M., Stevenson M., Rana T.M. Modulating HIV-1 replication by RNA interference directed against human transcription elongation factor SPT5. Retrovirology 2004, 1:46.
58. Chiu Y.L., Cao H., Jacque J.M., Stevenson M., Rana T.M. Inhibition of human immunodeficiency virus type 1 replication by RNA interference directed against human transcription elongation factor P-TEFb (CDK9/CyclinT1). J. Virol. 2004, 78:2517-2529.
59. Eekels J.J., Geerts D., Jeeninga R.E., Berkhout B. Long-term inhibition of HIV-1 replication with RNA interference against cellular co-factors. Antiviral Res. 2011, 89:43-53.
60. Westerhout E.M., Vink M., Haasnoot P.C., Das A.T., Berkhout B. A conditionally replicating HIV-based vector that stably expresses an antiviral shRNA against HIV-1 replication. Mol. Ther. 2006, 14:268-275.
61. Liu Y.P., Vink M.A., Westerink J.T., Ramirez de Arellano E., Konstantinova P., Ter Brake O., Berkhout B. Titers of lentiviral vectors encoding shRNAs and miRNAs are reduced by different mechanisms that require distinct repair strategies. RNA 2010, 16:1328-1339.
62. ter Brake O., Westerink J.T., Berkhout B. Lentiviral vector engineering for anti-HIV RNAi gene therapy. Methods Mol. Biol. 2010, 614:201-213.
63. Zhou D., Zhang J., Wang C., Bliesath J.R., He Q., Yu D., Li-He Z., Wong-Staal F. A method for detecting and preventing negative RNA interference in preparation of lentiviral vectors for siRNA delivery. RNA 2009, 15:732-740.
64. Poluri A., Sutton R.E. Titers of HIV-based vectors encoding shRNAs are reduced by a dicer-dependent mechanism. Mol. Ther. 2008, 16:378-386.
65. DiGiusto D.L., Krishnan A., Li L., Li H., Li S., Rao A., Mi S., Yam P., Stinson S., Kalos M., Alvarnas J., Lacey S.F., Yee J.K., Li M., Couture L., Hsu D., Forman S.J., Rossi J.J., Zaia J.A. RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma. Sci. Transl. Med. 2010, 2:36ra43.
66. Cummings I.W., Browne J.K., Salser W.A., Tyler G.V., Snyder R.L., Smolec J.M., Summers J. Isolation, characterization, and comparison of recombinant DNAs derived from genomes of human hepatitis B virus and woodchuck hepatitis virus. Proc. Natl. Acad. Sci. U.S.A. 1980, 77:1842-1846.
67. Summers J., O'Connell A., Millman I. Genome of hepatitis B virus: restriction enzyme cleavage and structure of DNA extracted from Dane particles. Proc. Natl. Acad. Sci. U.S.A. 1975, 72:4597-4601.
68. Tuttleman J.S., Pourcel C., Summers J. Formation of the pool of covalently closed circular viral DNA in hepadnavirus-infected cells. Cell 1986, 47:451-460.
69. Arbuthnot P., Thompson L.J. Harnessing the RNA interference pathway to advance treatment and prevention of hepatocellular carcinoma. World J. Gastroenterol. 2008, 14:1670-1681.
70. Chen Y., Cheng G., Mahato R.I. RNAi for treating hepatitis B viral infection. Pharm. Res. 2008, 25:72-86.
71. Arbuthnot P., Longshaw V., Naidoo T., Weinberg M.S. Opportunities for treating chronic hepatitis B and C virus infection using RNA interference. J. Viral Hepat. 2007, 14:447-459.
72. Romano P.R., McCallus D.E., Pachuk C.J. RNA interference-mediated prevention and therapy for hepatocellular carcinoma. Oncogene 2006, 25:3857-3865.
73. McCaffrey A.P., Nakai H., Pandey K., Huang Z., Salazar F.H., Xu H., Wieland S.F., Marion P.L., Kay M.A. Inhibition of hepatitis B virus in mice by RNA interference. Nat. Biotechnol. 2003, 21:639-644.
74. Weinberg M.S., Ely A., Barichievy S., Crowther C., Mufamadi S., Carmona S., Arbuthnot P. Specific inhibition of HBV replication in vitro and in vivo with expressed long hairpin RNA. Mol. Ther. 2007, 15:534-541.
75. Giladi H., Ketzinel-Gilad M., Rivkin L., Felig Y., Nussbaum O., Galun E. Small interfering RNA inhibits hepatitis B virus replication in mice. Mol. Ther. 2003, 8:769-776.
76. Hamasaki K., Nakao K., Matsumoto K., Ichikawa T., Ishikawa H., Eguchi K. Short interfering RNA-directed inhibition of hepatitis B virus replication. FEBS Lett. 2003, 543:51-54.
77. Klein C., Bock C.T., Wedemeyer H., Wustefeld T., Locarnini S., Dienes H.P., Kubicka S., Manns M.P., Trautwein C. Inhibition of hepatitis B virus replication in vivo by nucleoside analogues and siRNA. Gastroenterology 2003, 125:9-18.
78. Konishi M., Wu C.H., Wu G.Y. Inhibition of HBV replication by siRNA in a stable HBV-producing cell line. Hepatology 2003, 38:842-850.
79. Carmona S., Jorgensen M.R., Kolli S., Crowther C., Salazar F.H., Marion P.L., Fujino M., Natori Y., Thanou M., Arbuthnot P., Miller A.D. Controlling HBV replication in vivo by intravenous administration of triggered PEGylated siRNA-nanoparticles. Mol. Pharm. 2009, 6:706-717.
80. Carmona S., Ely A., Crowther C., Moolla N., Salazar F.H., Marion P.L., Ferry N., Weinberg M.S., Arbuthnot P. Effective inhibition of HBV replication in vivo by anti-HBx short hairpin RNAs. Mol. Ther. 2006, 13:411-421.
81. Kim Y.H., Lee J.H., Paik N.W., Rho H.M. RNAi-based knockdown of HBx mRNA in HBx-transformed and HBV-producing human liver cells. DNA Cell Biol. 2006, 25:412-417.
82. Ren X., Luo G., Xie Z., Zhou L., Kong X., Xu A. Inhibition of multiple gene expression and virus replication of HBV by stable RNA interference in 2.2.15 cells. J. Hepatol. 2006, 44:663-670.
83. Ren X.R., Zhou L.J., Luo G.B., Lin B., Xu A. Inhibition of hepatitis B virus replication in 2.2.15 cells by expressed shRNA. J. Viral Hepat. 2005, 12:236-242.
84. Shlomai A., Shaul Y. Inhibition of hepatitis B virus expression and replication by RNA interference. Hepatology 2003, 37:764-770.
85. Uprichard S.L., Boyd B., Althage A., Chisari F.V. Clearance of hepatitis B virus from the liver of transgenic mice by short hairpin RNAs. Proc. Natl. Acad. Sci. U.S.A. 2005, 102:773-778.
86. Chen Y., Du D., Wu J., Chan C.P., Tan Y., Kung H.F., He M.L. Inhibition of hepatitis B virus replication by stably expressed shRNA. Biochem. Biophys. Res. Commun. 2003, 311:398-404.
87. Keck K., Volper E.M., Spengler R.M., Long D.D., Chan C.Y., Ding Y., McCaffrey A.P. Rational design leads to more potent RNA interference against hepatitis B virus: factors effecting silencing efficiency. Mol. Ther. 2009, 17:538-547.
88. Sun D., Rosler C., Kidd-Ljunggren K., Nassal M. Quantitative assessment of the antiviral potencies of 21 shRNA vectors targeting conserved, including structured, hepatitis B virus sites. J. Hepatol. 2010, 52:817-826.
89. Grimm D., Streetz K.L., Jopling C.L., Storm T.A., Pandey K., Davis C.R., Marion P., Salazar F., Kay M.A. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006, 441:537-541.
90. Ely A., Naidoo T., Mufamadi S., Crowther C., Arbuthnot P. Expressed anti-HBV primary microRNA shuttles inhibit viral replication efficiently in vitro and in vivo. Mol. Ther. 2008, 16:1105-1112.
91. Ely A., Arbuthnot P. Silencing hepatitis B virus replication with antiviral pri-miR shuttles generated from liver-specific Pol II promoter. Gene Silencing: Theory 2010, Nova Publishers, Techniques and Applications. A.J. Catalano (Ed.).
92. Ely A., Naidoo T., Arbuthnot P. Efficient silencing of gene expression with modular trimeric Pol II expression cassettes comprising microRNA shuttles. Nucleic Acids Res. 2009, 37:e91.
93. Arbuthnot P., Ely A., Weinberg M.S. Hepatic delivery of RNA interference activators for therapeutic application. Curr. Gene Ther. 2009, 9:91-103.
94. Mowa M.B., Crowther C., Arbuthnot P. Therapeutic potential of adenoviral vectors for delivery of expressed RNAi activators. Expert Opin. Drug Deliv. 2010, 7:1373-1385.
95. Waddington S.N., McVey J.H., Bhella D., Parker A.L., Barker K., Atoda H., Pink R., Buckley S.M., Greig J.A., Denby L., Custers J., Morita T., Francischetti I.M., Monteiro R.Q., Barouch D.H., van Rooijen N., Napoli C., Havenga M.J., Nicklin S.A., Baker A.H. Adenovirus serotype 5 hexon mediates liver gene transfer. Cell 2008, 132:397-409.
96. Crowther C., Ely A., Hornby J., Mufamadi S., Salazar F., Marion P., Arbuthnot P. Efficient inhibition of hepatitis B virus replication in vivo, using polyethylene glycol-modified adenovirus vectors. Hum. Gene Ther. 2008, 19:1325-1331.
97. Ferrari F.K., Samulski T., Shenk T., Samulski R.J. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 1996, 70:3227-3234.
98. Sipo I., Fechner H., Pinkert S., Suckau L., Wang X., Weger S., Poller W. Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction. Gene Ther. 2007, 14:1319-1329.
99. Wu Z., Asokan A., Samulski R.J. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol. Ther. 2006, 14:316-327.
100. Grimm D., Pandey K., Nakai H., Storm T.A., Kay M.A. Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J. Virol. 2006, 80:426-439.
101. Chen C.C., Ko T.M., Ma H.I., Wu H.L., Xiao X., Li J., Chang C.M., Wu P.Y., Chen C.H., Han J.M., Yu C.P., Jeng K.S., Hu C.P., Tao M.H. Long-term inhibition of hepatitis B virus in transgenic mice by double-stranded adeno-associated virus 8-delivered short hairpin RNA. Gene Ther. 2007, 14:11-19.
102. Chen C.C., Sun C.P., Ma H.I., Fang C.C., Wu P.Y., Xiao X., Tao M.H. Comparative study of anti-hepatitis B virus RNA interference by double-stranded adeno-associated virus serotypes 7, 8, and 9. Mol. Ther. 2009, 17:352-359.
103. Birraux J., Wildhaber B.E., Jond C., Belli D.C., Menzel O. Ex vivo hepatocyte gene therapy: increased biosafety protocol for transduction in suspension with lentiviral vectors and immediate transplantation. Transplantation 2010, 89:127-129.
104. Menzel O., Birraux J., Wildhaber B.E., Jond C., Lasne F., Habre W., Trono D., Nguyen T.H., Chardot C. Biosafety in ex vivo gene therapy and conditional ablation of lentivirally transduced hepatocytes in nonhuman primates. Mol. Ther. 2009, 17:1754-1760.
105. Birraux J., Menzel O., Wildhaber B., Jond C., Nguyen T.H., Chardot C. A step toward liver gene therapy: efficient correction of the genetic defect of hepatocytes isolated from a patient with Crigler-Najjar syndrome type 1 with lentiviral vectors. Transplantation 2009, 87:1006-1012.
106. Nguyen T.H., Birraux J., Wildhaber B., Myara A., Trivin F., Le Coultre C., Trono D., Chardot C. Ex vivo lentivirus transduction and immediate transplantation of uncultured hepatocytes for treating hyperbilirubinemic Gunn rat. Transplantation 2006, 82:794-803.
107. Ghany M.G., Strader D.B., Thomas D.L., Seeff L.B. Diagnosis, management, and treatment of hepatitis C: an update. Hepatology 2009, 49:1335-1374.
108. Bartenschlager R., Frese M., Pietschmann T. Novel insights into hepatitis C virus replication and persistence. Adv. Virus Res. 2004, 63:71-180.
109. Bartenschlager R., Lohmann V. Replication of hepatitis C virus. J. Gen. Virol. 2000, 81:1631-1648.
110. Pan Q., Henry S.D., Metselaar H.J., Scholte B., Kwekkeboom J., Tilanus H.W., Janssen H.L., van der Laan L.J. Combined antiviral activity of interferon-alpha and RNA interference directed against hepatitis C without affecting vector delivery and gene silencing. J. Mol. Med. 2009, 87:713-722.
111. Wang Y., Kato N., Jazag A., Dharel N., Otsuka M., Taniguchi H., Kawabe T., Omata M. Hepatitis C virus core protein is a potent inhibitor of RNA silencing-based antiviral response. Gastroenterology 2006, 130:883-892.
112. Yokota T., Sakamoto N., Enomoto N., Tanabe Y., Miyagishi M., Maekawa S., Yi L., Kurosaki M., Taira K., Watanabe M., Mizusawa H. Inhibition of intracellular hepatitis C virus replication by synthetic and vector-derived small interfering RNAs. EMBO Rep. 2003, 4:602-608.
113. Takigawa Y., Nagano-Fujii M., Deng L., Hidajat R., Tanaka M., Mizuta H., Hotta H. Suppression of hepatitis C virus replicon by RNA interference directed against the NS3 and NS5B regions of the viral genome. Microbiol. Immunol. 2004, 48:591-598.
114. Ray R.B., Kanda T. Inhibition of HCV replication by small interfering RNA. Methods Mol. Biol. 2009, 510:251-262.
115. Shin D., Lee H., Kim S.I., Yoon Y., Kim M. Optimization of linear double-stranded RNA for the production of multiple siRNAs targeting hepatitis C virus. RNA 2009, 15:898-910.
116. Yang X., Haurigot V., Zhou S., Luo G., Couto L.B. Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microRNA cluster. Hepatology 2010, 52:1877-1887.
117. Randall G., Panis M., Cooper J.D., Tellinghuisen T.L., Sukhodolets K.E., Pfeffer S., Landthaler M., Landgraf P., Kan S., Lindenbach B.D., Chien M., Weir D.B., Russo J.J., Ju J., Brownstein M.J., Sheridan R., Sander C., Zavolan M., Tuschl T., Rice C.M. Cellular cofactors affecting hepatitis C virus infection and replication. Proc. Natl. Acad. Sci. U.S.A. 2007, 104:12884-12889.
118. Tai A.W., Benita Y., Peng L.F., Kim S.S., Sakamoto N., Xavier R.J., Chung R.T. A functional genomics screen identifies cellular cofactors of hepatitis C virus replication. Cell Host Microbe 2009, 5:298-307.
119. Jopling C.L., Yi M., Lancaster A.M., Lemon S.M., Sarnow P. Modulation of hepatitis C virus RNA abundance by a liver-specific MicroRNA. Science 2005, 309:1577-1581.
120. Lanford R.E., Hildebrandt-Eriksen E.S., Petri A., Persson R., Lindow M., Munk M.E., Kauppinen S., Orum H. Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science 2009, 327:198-201.
121. Henry S.D., van der Wegen P., Metselaar H.J., Tilanus H.W., Scholte B.J., van der Laan L.J. Simultaneous targeting of HCV replication and viral binding with a single lentiviral vector containing multiple RNA interference expression cassettes. Mol. Ther. 2006, 14:485-493.
122. Thimme R., Bukh J., Spangenberg H.C., Wieland S., Pemberton J., Steiger C., Govindarajan S., Purcell R.H., Chisari F.V. Viral and immunological determinants of hepatitis C virus clearance, persistence, and disease. Proc. Natl. Acad. Sci. U.S.A. 2002, 99:15661-15668.
123. Guevin C., Lamarre A., Labonte P. Novel HCV replication mouse model using human hepatocellular carcinoma xenografts. Antiviral Res. 2009, 84:14-22.
124. Zhu Q., Weiner A.J. A hepatitis C virus xenograft mouse efficacy model. Methods Mol. Biol. 2009, 511:323-331.
125. Dengue Fever Fact Sheet, Centers for Disease Control and Prevention (CDC) 2005.
126. Subramanya S., Kim S.S., Abraham S., Yao J., Kumar M., Kumar P., Haridas V., Lee S.K., Shultz L.D., Greiner D., Shankar M.N.P. Targeted delivery of small interfering RNA to human dendritic cells to suppress dengue virus infection and associated proinflammatory cytokine production. J. Virol. 2010, 84:2490-2501.
127. Zhang W., Singam R., Hellermann G., Kong X., Juan H.S., Lockey R.F., Wu S.J., Porter K., Mohapatra S.S. Attenuation of dengue virus infection by adeno-associated virus-mediated siRNA delivery. Genet. Vaccines Ther. 2004, 2. 10.1186/1479-0556-1182-1188.
128. Adelman Z.N., Sanchez-Vargas I., Travanty E.A., Carlson J.O., Beaty B.J., Blair C.D., Olson K.E. RNA silencing of dengue virus type 2 replication in transformed C6/36 mosquito cells transcribing an inverted-repeat RNA derived from the virus genome. J. Virol. 2002, 76:12925-12933.
129. Travanty E.A., Adelman Z.N., Franz A.W., Keene K.M., Beaty B.J., Blair C.D., James A.A., Olson K.E. Using RNA interference to develop dengue virus resistance in genetically modified Aedes aegypti. Insect Biochem. Mol. Biol. 2004, 34:607-613.
130. Franz A.W., Sanchez-Vargas I., Adelman Z.N., Blair C.D., Beaty B.J., James A.A., Olson K.E. Engineering RNA interference-based resistance to dengue virus type 2 in genetically modified Aedes aegypti. Proc. Natl. Acad. Sci. U.S.A. 2006, 103:4198-4203.
131. Franz A.W., Sanchez-Vargas I., Piper J., Smith M.R., Khoo C.C., James A.A., Olson K.E. Stability and loss of a virus resistance phenotype over time in transgenic mosquitoes harbouring an antiviral effector gene. Insect Mol. Biol. 2009, 18:661-672.
132. Grimm D., Wang L., Lee J.S., Schurmann N., Gu S., Borner K., Storm T.A., Kay M.A. Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J. Clin. Invest. 2010, 120:3106-3119.
133. Witting S.R., Brown M., Saxena R., Nabinger S., Morral N. Helper-dependent adenovirus-mediated short hairpin RNA expression in the liver activates the interferon response. J. Biol. Chem. 2008, 283:2120-2128.
134. Schmidt C. RNAi momentum fizzles as pharma shifts priorities. Nat. Biotechnol. 2011, 29:93-94.