Delivery of siRNA and siRNA expression constructs to adult mammals by hydrodynamic intravascular injection

Methods in Enzymology

Volumn 392, Issue , 2005, Pages 336-350

  Lewis, David L ^a   Wolff, Jon A ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

PLASMID DNA; SMALL INTERFERING RNA;

BILE DUCT; BLOOD VESSEL; BODY WEIGHT; CAVA VEIN; CELL TYPE; DRUG DELIVERY SYSTEM; GENE CONSTRUCT; GENE EXPRESSION; GENE FUNCTION; GENE TARGETING; HYDRODYNAMICS; LIVER; LIVER CELL; MAMMAL; MOUSE STRAIN; NONHUMAN; PORTAL VEIN; PRESSURE; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN TRANSPORT; REVIEW; RNA INTERFERENCE; TAIL;

MAMMALIA; PRIMATES;

EID: 11844262129     PISSN: 00766879     EISSN: None     Source Type: Book Series    
DOI: 10.1016/S0076-6879(04)92020-4     Document Type: Article

References (39)

1. Aza-Blanc P., Cooper C.L., Wagner K., Batalov S., Deveraux Q.L., Cooke M.P. Identification of modulators of TRAIL-induced apoptosis via RNAi-based phenotypic screening. Mol. Cell. 12:2003;627-637
2. Berns K., Hijmans E.M., Mullenders J., Brummelkamp T.R., Velds A., Heimerikx M., Kerkhoven R.M., Madiredjo M., Nijkamp W., Weigelt B., Agami R., Ge W., Cavet G., Linsley P.S., Beijersbergen R.L., Bernards R. A large-scale RNAi screen in human cells identifies new components of the p53 pathway. Nature. 428:2004;431-437
3. Boutla A., Delidakis C., Livadaras I., Tsagris M., Tabler M. Short 5′-phosphorylated double-stranded RNAs induce RNA interference in Drosophila. Curr. Biol. 11:2001;1776-1780
4. Brummelkamp T.R., Nijman S.M., Dirac A.M., Bernards R. Loss of the cylindromatosis tumour suppressor inhibits apoptosis by activating NF-kappaB. Nature. 424:2003;797-801
5. Budker V., Budker T., Zhang G., Subbotin V., Loomis A., Wolff J.A. Hypothesis: Naked plasmid DNA is taken up by cells in vivo by a receptor-mediated process. J. Gene Med. 2:2000;76-88
6. Budker V., Zhang G., Danko I., Williams P., Wolff J. The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther. 5:1998;272-276
7. Budker V., Zhang G., Knechtle S., Wolff J.A. Naked DNA delivered intraportally expresses efficiently in hepatocytes. Gene Ther. 3:1996;593-598
8. Caplen N.J., Parrish S., Imani F., Fire A., Morgan R.A. Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems. Proc. Natl. Acad. Sci. USA. 98:2001;9742-9747
9. Caudy A.A., Ketting R.F., Hammond S.M., Denli A.M., Bathoorn A.M., Tops B.B., Silva J.M., Myers M.M., Hannon G.J., Plasterk R.H. A micrococcal nuclease homologue in RNAi effector complexes. Nature. 425:2003;411-414
10. Eastman S.J., Baskin K.M., Hodges B.L., Chu Q., Gates A., Dreusicke R., Anderson S., Scheule R.K. Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA. Hum. Gene Ther. 13:2002;2065-2077
11. Elbashir S.M., Lendeckel W., Tuschl T. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev. 15:2001a;188-200
12. 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. 411:2001b;494-498
13. Fire A., Xu S., Montgomery M.K., Kostas S.A., Driver S.E., Mello C.C. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 391:1998;806-811
14. 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. 8:2003;769-776
15. Hammond S.M., Bernstein E., Beach D., Hannon G.J. An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature. 404:2000;293-296
16. Hickman M.A., Malone R.W., Lehmann-Bruinsma K., Sih T.R., Knoell D., Szoka F.C., Walzem R., Carlson D.M., Powell J.S. Gene expression following direct injection of DNA into liver. Hum. Gene Ther. 5:1994;1477-1483
17. Hutvagner G., Zamore P.D. A microRNA in a multiple-turnover RNAi enzyme complex. Science. 297:2002;2056-2060
18. Kawabata K., Takakura Y., Hashida M. The fate of plasmid DNA after intravenous injection in mice: Involvement of scavenger receptors in its hepatic uptake. Pharm. Res. 12:1995;825-830
19. Kobayashi N., Kuramoto T., Yamaoka K., Hashida M., Takakura Y. Hepatic uptake and gene expression mechanisms following intravenous administration of plasmid DNA by conventional and hydrodynamics-based procedures. J. Pharmacol. Exp. Ther. 297:2001;853-860
20. Lewis D.L., Hagstrom J.E., Loomis A.G., Wolff J.A., Herweijer H. Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat. Genet. 32:2002;107-108
21. Liu F., Song Y., Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. 6:1999;1258-1266
22. Ludtke J.J., Zhang G., Sebestyen M.G., Wolff J.A. A nuclear localization signal can enhance both the nuclear transport and expression of 1 kb DNA. J. Cell Sci. 112:1999;2033-2041
23. Martinez J., Patkaniowska A., Urlaub H., Luhrmann R., Tuschl T. Single-stranded antisense siRNAs guide target RNA cleavage in RNAi. Cell. 110:2002;563-574
24. McCaffrey A.P., Meuse L., Pham T.T., Conklin D.S., Hannon G.J., Kay M.A. RNA interference in adult mice. Nature. 418:2002;38-39
25. 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. 21:2003;639-644
26. McManus M.T., Sharp P.A. Gene silencing in mammals by small interfering RNAs. Nat. Rev. Genet. 3:2002;737-747
27. Nykanen A., Haley B., Zamore P.D. ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell. 107:2001;309-321
28. Paddison P.J., Silva J.M., Conklin D.S., Schlabach M., Li M., Aruleba S., Balija V., O'Shaughnessy A., Gnoj L., Scobie K., Chang K., Westbrook T., Cleary M., Sachidanandam R., McCombie W.R., Elledge S.J., Hannon G.J. A resource for large-scale RNA-interference-based screens in mammals. Nature. 428:2004;427-431
29. Parrish S., Fleenor J., Xu S., Mello C., Fire A. Functional anatomy of a dsRNA trigger. Differential requirement for the two trigger strands in RNA interference. Mol. Cell. 6:2000;1077-1087
30. Song E., Lee S.K., Wang J., Ince N., Ouyang N., Min J., Chen J., Shankar P., Lieberman J. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat. Med. 9:2003;347-351
31. Stark G.R., Kerr I.M., Williams B.R., Silverman R.H., Schreiber R.D. How cells respond to interferons. Annu. Rev. Biochem. 67:1998;227-264
32. Zamore P.D., Tuschl T., Sharp P.A., Bartel D.P. RNAi: Double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell. 101:2000;25
33. Zender L., Hutker S., Liedtke C., Tillmann H.L., Zender S., Mundt B., Waltemathe M., Gosling T., Flemming P., Malek N.P., Trautwein C., Manns M.P., Kuhnel F., Kubicka S. Caspase 8 small interfering RNA prevents acute liver failure in mice. Proc. Natl. Acad. Sci. USA. 100:2003;7797-7802
34. Zhang G., Budker V., Williams P., Hanson K., Wolff J.A. Surgical procedures for intravascular delivery of plasmid DNA to organs. Methods Enzymol. 346:2002;125-133
35. Zhang G., Budker V., Williams P., Subbotin V., Wolff J.A. Efficient expression of naked DNA delivered intraarterially to limb muscles of nonhuman primates. Hum. Gene Ther. 12:2001;427-438
36. Zhang G., Budker V., Wolff J.A. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum. Gene Ther. 10:1999;1735-1737
37. Zhang G., Gao X., Song Y.K., Vollmer R., Stolz D.B., Gasiorowski J.Z., Dean D.A., Liu D. Hydroporation as the mechanism of hydrodynamic delivery. Gene Ther. 11:2004;675-682
38. Zhang G., Vargo D., Budker V., Armstrong N., Knechtle S., Wolff J.A. Expression of naked plasmid DNA injected into the afferent and efferent vessels of rodent and dog livers. Hum. Gene Ther. 8:1997;1763-1772
39. Zheng L., Liu J., Batalov S., Zhou D., Orth A., Ding S., Schultz P.G. An approach to genomewide screens of expressed small interfering RNAs in mammalian cells. Proc. Natl. Acad. Sci. USA. 101:2004;135-140