RNAi and small interfering RNAs in human disease therapeutic applications

Trends in Biotechnology

Volumn 28, Issue 11, 2010, Pages 570-579

  Lares, Monica R ^a   Rossi, John J ^a   Ouellet, Dominique L ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CLINICAL APPLICATION; HUMAN CELLS; HUMAN DISEASE; SMALL INTERFERING RNA; THERAPEUTIC APPLICATION; THERAPEUTIC MODALITY; VIRAL INFECTIONS; VIRAL VECTORS;

RNA;

GENE EXPRESSION;

AGN 211745; AT 027; BEVASIRANIB; CEQ 508; EZN 2968; EZN 3042; LIGAND; LIPID; NANOPARTICLE; PF 4523655; POLYMER; PRO 040201; QPI 1007; SMALL INTERFERING RNA; SPC 2996; SPC 3649; SPC 4955; SURVIVIN; TD 1010; UNCLASSIFIED DRUG; VIRUS VECTOR;

ADENOMATOUS POLYP; CHRONIC LYMPHATIC LEUKEMIA; CLINICAL TRIAL; DRUG DELIVERY SYSTEM; GENE EXPRESSION; GENE TARGETING; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; HYPERCHOLESTEROLEMIA; IMMUNE RESPONSE; NONHUMAN; OPTIC NERVE INJURY; PRIORITY JOURNAL; RETINA MACULA AGE RELATED DEGENERATION; RETINA MACULA EDEMA; REVIEW; RNA INTERFERENCE; SOLID TUMOR;

BIOLOGICAL PRODUCTS; GENE KNOCKDOWN TECHNIQUES; GENE THERAPY; HUMANS; RNA, SMALL INTERFERING;

EID: 77957820261     PISSN: 01677799     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.tibtech.2010.07.009     Document Type: Review

References (78)

1. Elbashir S.M., et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411:494-498.
2. Kim D.H., et al. Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy. Nat. Biotechnol. 2005, 23:222-226.
3. Chang C.I., et al. Dual-target gene silencing by using long, synthetic siRNA duplexes without triggering antiviral responses. Mol. Cells 2009, 27:689-695.
4. Elbashir S.M., et al. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev. 2001, 15:188-200.
5. Weinberg M.S., et al. An RNA targeted to the HIV-1 LTR promoter modulates indiscriminate off-target gene activation. Nucleic Acids Res. 2007, 35:7303-7312.
6. Bartel D.P. MicroRNAs: target recognition and regulatory functions. Cell 2009, 136:215-233.
7. Siomi H., Siomi M.C. Posttranscriptional regulation of microRNA biogenesis in animals. Mol. Cell 2010, 38:323-332.
8. Jopling C.L., et al. Modulation of hepatitis C virus RNA abundance by a liver-specific MicroRNA. Science 2005, 309:1577-1581.
9. Naito Y., et al. Optimal design and validation of antiviral siRNA for targeting HIV-1. Retrovirology 2007, 4:80.
10. Gitlin L., et al. Short interfering RNA confers intracellular antiviral immunity in human cells. Nature 2002, 418:430-434.
11. Lu X., et al. Antisense-mediated inhibition of human immunodeficiency virus (HIV) replication by use of an HIV type 1-based vector results in severely attenuated mutants incapable of developing resistance. J. Virol. 2004, 78:7079-7088.
12. Zhang J., et al. Therapeutic potential of RNA interference against cellular targets of HIV infection. Mol. Biotechnol. 2007, 37:225-236.
13. Samson M., et al. Resistance to HIV-1 infection in Caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature 1996, 382:722-725.
14. von Eije K.J., et al. Human immunodeficiency virus type 1 escape is restricted when conserved genome sequences are targeted by RNA interference. J. Virol. 2008, 82:2895-2903.
15. Schopman N.C., et al. Anticipating and blocking HIV-1 escape by second generation antiviral shRNAs. Retrovirology 2010, 7:52.
16. Puerta-Fernandez E., et al. Inhibition of HIV-1 replication by RNA targeted against the LTR region. AIDS 2005, 19:863-870.
17. Weinberg M.S., et al. The antisense strand of small interfering RNAs directs histone methylation and transcriptional gene silencing in human cells. RNA 2006, 12:256-262.
18. Moses J., et al. Intended transcriptional silencing with siRNA results in gene repression through sequence-specific off-targeting. RNA 2010, 16:430-441.
19. Stein C.A., et al. Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents. Nucleic Acids Res. 2010, 38:e3.
20. Shukla S., et al. Exploring chemical modifications for siRNA therapeutics: a structural and functional outlook. Chem. Med. Chem. 2010, 5:328-349.
21. Judge A.D., et al. Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo. Mol. Ther. 2006, 13:494-505.
22. Czauderna F., et al. Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res. 2003, 31:2705-2716.
23. McNamara J.O., et al. Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat. Biotech. 2006, 24:1005-1015.
24. Dassie J.P., et al. Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors. Nat. Biotechnol. 2009, 27:839-849.
25. Zhou J., et al. Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells. Nucleic Acids Res. 2009, 37:3094-3109.
26. Kumar P., et al. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 2008, 134:577-586.
27. Song E., et al. Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat. Biotechnol. 2005, 23:709-717.
28. Peer D., et al. Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 2008, 319:627-630.
29. Soutschek J., et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 2004, 432:173-178.
30. Felgner P.L., Ringold G.M. Cationic liposome-mediated transfection. Nature 1989, 337:387-388.
31. Morrissey D.V., et al. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat. Biotechnol. 2005, 23:1002-1007.
32. Frank-Kamenetsky M., et al. Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Proc. Natl. Acad. Sci. U. S. A. 2008, 105:11915-11920.
33. Sonoke S., et al. Tumor regression in mice by delivery of Bcl-2 small interfering RNA with pegylated cationic liposomes. Cancer Res. 2008, 68:8843-8851.
34. Leng Q., et al. Histidine-lysine peptides as carriers of nucleic acids. Drug News Perspect. 2007, 20:77-86.
35. Wu Y., et al. The investigation of polymer-siRNA nanoparticle for gene therapy of gastric cancer in vitro. Int. J. Nanomed. 2010, 5:129-136.
36. Azuma K., et al. Anti-tumor effect of small interfering RNA targeting the androgen receptor in human androgen-independent prostate cancer cells. Biochem. Biophys. Res. Commun. 2010, 391:1075-1079.
37. Kim S.H., et al. VEGF siRNA delivery system using arginine-grafted bioreducible poly(disulfide amine). Mol. Pharmaceut. 2008, 6:718-726.
38. Davis M.E., et al. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 2010, 464:1067-1070.
39. Cavazzana-Calvo M., et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000, 288:669-672.
40. Miyoshi H., et al. Development of a self-inactivating lentivirus vector. J. Virol. 1998, 72:8150-8157.
41. 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.
42. Hacein-Bey-Abina S., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003, 302:415-419.
43. Hoshang U., John R. Tat-regulated expression of RNA interference: triggers for the treatment of HIV infection. Curr. HIV/AIDS Rep. 2008, 5:40-43.
44. Yam P.Y., et al. Design of HIV vectors for efficient gene delivery into human hematopoietic cells. Mol. Ther. 2002, 5:479-484.
45. Kamata M., et al. Generation of human induced pluripotent stem cells bearing an anti-HIV transgene by a lentiviral vector carrying an internal murine leukemia virus promoter. Hum. Gene Ther. 2010, 10.1089/hum.2010.050.
46. Gijsbers R., et al. LEDGF hybrids efficiently retarget lentiviral integration into heterochromatin. Mol. Ther. 2010, 18:552-560.
47. Baoliang G., et al. Lentivirus-mediated small interfering RNA targeting VEGF-C inhibited tumor lymphangiogenesis and growth in breast carcinoma. Anat. Rec. (Hoboken) 2009, 292:633-639.
48. Michelfelder S., Trepel M. Adeno-associated viral vectors and their redirection to cell-type specific receptors. Adv. Genet. 2009, 67:29-60.
49. Nayak S., Herzog R.W. Progress and prospects: immune responses to viral vectors. Gene Ther. 2010, 17:295-304.
50. Kim S.M., et al. Multiple shRNAs driven by U6 and CMV promoter show enhanced of antiviral effects against foot-and-mouth disease virus. Antiviral Res. 2010, 87:307-317.
51. Nakai H., et al. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J. Virol. 2005, 79:214-224.
52. Beer S., et al. Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice. Mol. Ther. 2010, 18:161-170.
53. Siolas D., et al. Synthetic shRNAs as potent RNAi triggers. Nat. Biotechnol. 2005, 23:227-231.
54. Giering J.C., et al. Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Mol. Ther. 2008, 16:1630-1636.
55. Grimm D., et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006, 441:537-541.
56. Aagaard L.A., et al. Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs. Gene Ther. 2008, 15:1536-1549.
57. Escors D., Breckpot K. Lentiviral vectors in gene therapy: their current status and future potential. Arch. Immunol. Ther. Exp. (Warsz) 2010, 58:107-119.
58. Ehsani A., et al. Rational design of micro-RNA-like bifunctional siRNAs targeting HIV and the HIV coreceptor CCR5. Mol. Ther. 2010, 18:796-802.
59. Saetrom P. Predicting the efficacy of short oligonucleotides in antisense and RNAi experiments with boosted genetic programming. Bioinformatics 2004, 20:3055-3063.
60. Liu Y.P., et al. Design of extended short hairpin RNAs for HIV-1 inhibition. Nucleic Acids Res. 2007, 35:5683-5693.
61. Liu Y.P., et al. Combinatorial RNAi against HIV-1 using extended short hairpin RNAs. Mol. Ther. 2009, 17:1712-1723.
62. Barichievy S., et al. 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.
63. Saayman S., et al. Deriving four functional anti-HIV siRNAs from a single Pol III-generated transcript comprising two adjacent long hairpin RNA precursors. Nucleic Acids Res. 2010, 10.1093/nar/gkq460.
64. Bauer M., et al. Prevention of interferon-stimulated gene expression using microRNA-designed hairpins. Gene Ther. 2009, 16:142-147.
65. Liu Y.P., et al. Inhibition of HIV-1 by multiple siRNAs expressed from a single microRNA polycistron. Nucleic Acids Res. 2008, 36:2811-2824.
66. Li M.J., et al. Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy. Mol. Ther. 2005, 12:900-909.
67. DiGiusto D.L., et al. 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.
68. Unwalla H.J., et al. 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.
69. Takeda K., Akira S. Toll-like receptors in innate immunity. Int. Immunol. 2005, 17:1-14.
70. Marques J.T., Williams B.R. Activation of the mammalian immune system by siRNAs. Nat. Biotechnol. 2005, 23:1399-1405.
71. Poeck H., et al. 5′-Triphosphate-siRNA: turning gene silencing and Rig-I activation against melanoma. Nat. Med. 2008, 14:1256-1263.
72. Pastor F., et al. Induction of tumour immunity by targeted inhibition of nonsense-mediated mRNA decay. Nature 2010, 465:227-230.
73. Gantier M.P., et al. Rational design of immunostimulatory siRNAs. Mol. Ther. 2010, 18:785-795.
74. Laursen M.B., et al. Utilization of unlocked nucleic acid (UNA) to enhance siRNA performance in vitro and in vivo. Mol. Biosyst. 2010, 6:862-870.
75. Keates A.C., et al. Cequent Pharmaceuticals, Inc.: the biological pitcher for RNAi therapeutics. Pharmacogenomics 2007, 8:867-871.
76. McCarthy E.F. The toxins of William B. Coley and the treatment of bone and soft-tissue sarcomas. Iowa Orthop. J 2006, 26:154-158.
77. MacDiarmid J.A., et al. Sequential treatment of drug-resistant tumors with targeted minicells containing siRNA or a cytotoxic drug. Nat. Biotech. 2009, 27:643-651.
78. Yang N., et al. Oral administration of attenuated S. typhimurium carrying shRNA-expressin vectors as a cancer therapeutic. Cancer Biol. Ther. 2008, 7:147-153.