Challenges for RNAi in vivo

Trends in Biotechnology

Volumn 22, Issue 8, 2004, Pages 390-394

  Paroo, Zain ^a   Corey, David R ^a  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CELL CULTURE; RNA;

GENE FUNCTION; GENE SILENCING;

GENETIC ENGINEERING;

ANTISENSE OLIGODEOXYNUCLEOTIDE; SMALL INTERFERING RNA;

BIOLUMINESCENCE; CHEMICAL MODIFICATION; DNA STRAND; GENE SILENCING; GENETIC TRANSFECTION; PRIORITY JOURNAL; REVIEW; RNA ANALYSIS; RNA STRUCTURE; THERMOSTABILITY;

MAMMALIA;

EID: 3342903336     PISSN: 01677799     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.tibtech.2004.06.004     Document Type: Article

References (35)

1. Denli A.M., Hannon G.J. RNAi: an ever-growing puzzle. Trends Biochem. Sci. 28:2003;196-201
2. Elbashir S.M., et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 411:2001;494-498
3. Braasch D.A., et al. RNA interference in mammalian cells by chemically-modified RNA. Biochemistry. 42:2003;7967-7975
4. Hough S.R., et al. Why RNAi makes sense. Nat. Biotechnol. 21:2003;731-732
5. Hogrefe R.I. An antisense oligonucleotide primer. Antisense Nucleic Acid Drug Dev. 9:1999;351-357
6. Crooke S.T. Evaluating the mechanism of action of antiproliferative antisense drugs. Antisense Nucleic Acid Drug Dev. 10:2000;123-126
7. Sands H., et al. Biodistribution and metabolism of internally 3H-labeled oligonucleotides. I. Comparison of a phosphodiester and a phosphorothioate. Mol. Pharmacol. 45:1994;932-943
8. Geary R.S., et al. Pharmacokinetics of phosphorothioate antisense oligonucleotides. Curr. Opin. Investig. Drugs. 2:2001;562-573
9. Geary R.S., et al. Pharmacokinetic properties of 2′-O-(2- methoxyethyl)-modified antisense oligonucleotides analogs in rats. J. Pharmacol. Exp. Ther. 296:2001;890-897
10. Cho-Chung Y.S., Becker K.G. A genome-wide view of antisense. Nat. Biotechnol. 21:2003;492
11. Jackson A.L., et al. Expression profiling reveals off-target gene regulation by RNAi. Nat. Biotechnol. 21:2003;635-637
12. Semizarov D., et al. Specificity of short interfering RNA determined through gene expression signatures. Proc. Natl. Acad. Sci. U. S. A. 100:2003;6347-6352
13. Krieg A.M. CpG motifs in bacterial DNA and their immune effects. Annu. Rev. Immunol. 20:2002;709-760
14. Sledz C.A., et al. Activation of the interferon system by short-interfering RNAs. Nat. Cell Biol. 5:2003;834-839
15. Kim D.H., et al. Interferon induction by siRNAs and ssRNAs synthesized by phage polymerase. Nat. Biotechnol. 22:2004;321-325
16. Editorial. Wither RNAi? Nat. Cell Biol. 5:2003;489-490
17. McCaffrey A.P., et al. RNA interference in adult mice. Nature. 418:2002;38-39
18. Lewis D.L., et al. Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat. Genet. 32:2002;107-108
19. Zhang H., et al. Reduction of liver Fas expression by an antisense oligonucleotide protects mice from fulminant hepatitis. Nat. Biotechnol. 18:2000;862-867
20. Song E., et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat. Med. 9:2003;347-351
21. Zender L., et al. Caspase 8 small interfering RNA prevents acute liver failure in mice. Proc. Natl. Acad. Sci. U. S. A. 100:2003;7797-7802
22. Sorensen D.R., et al. Gene silencing by systemic delivery of synthetic siRNAs in adult mice. J. Mol. Biol. 327:2003;761-766
23. Dean N.M., McKay R. Inhibition of protein kinase C-alpha expression in mice after systemic administration of phosphorothioate antisense oligodeoxynucleotides. Proc. Natl. Acad. Sci. U. S. A. 91:1994;11762-11766
24. Dow S.W., et al. Lipid-DNA complexes induce potent activation of innate immune responses and antitumor activity when administered intravenously. J. Immunol. 163:1999;1552-1561
25. Tousignant J.D., et al. Comprehensive analysis of the acute toxicities induced by systemic administration of cationic lipid:plasmid DNA complexes in mice. Hum. Gene Ther. 11:2000;2493-2513
26. Braasch D.A., et al. Biodistribution of native and phosphorothioate siRNA. Bioorg. Med. Chem. Lett. 14:2004;1139-1143
27. Agrawal S., et al. Toxicologic effects of an oligodeoxynucleotide phosphorothioate and its analogs following intravenous administration in rats. Antisense Nucleic Acid Drug Dev. 7:1997;575-584
28. Hough S.R., et al. Why RNAi makes sense. Nat. Biotechnol. 21:2003;731-732
29. Harborth J., et al. Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing. Antisense Nucleic Acid Drug Dev. 13:2003;83-105
30. Chiu Y.L., Rana T.M. siRNA function in RNAi: a chemical modification analysis. RNA. 9:2003;1034-1048
31. Czauderna F., et al. Structural variations and stabilizing modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res. 31:2003;2705-2716
32. Amarzguioui M., et al. Tolerance for mutations and chemical modifications in an siRNA. Nucleic Acids Res. 31:2003;589-595
33. Khvorova A., et al. Functional siRNAs and miRNAs exhibit strand bias. Cell. 115:2003;209-216
34. Schwarz D.S., et al. Asymmetry in the assembly of the RNAi enzyme complex. Cell. 115:2003;199-208
35. Hamada M., et al. Effects on RNA interference in gene expression (RNAi) in cultured mammalian cells of mismatches and the introduction of chemical modifications at the 3′-ends of siRNAs. Antisense Nucleic Acid Drug Dev. 12:2002;301-309