Lipidic carriers of siRNA: Differences in the formulation, cellular uptake, and delivery with plasmid DNA

Biochemistry

Volumn 43, Issue 42, 2004, Pages 13348-13356

  Spagnou, Sebastien ^a   Miller, Andrew D ^a,b   Keller, Michael ^a  

^a IC Vec Ltd   (United Kingdom)

^b IMPERIAL COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BIOSYNTHESIS; CELL CULTURE; CHOLESTEROL; DNA SEQUENCES; GENES; LIPIDS; MOLECULAR BIOLOGY; PARTICLE SIZE ANALYSIS;

GENE EXPRESSION; GENE SILENCING; MOLAR RATIOS; RNA INTERFERENCE (RNAI);

RNA;

CELL PROTEIN; DIOLEOYL LEVO ALPHA PHOSPHATIDYLETHANOLAMINE; DOUBLE STRANDED RNA; GENOMIC RNA; LIPID; LIPOFECTAMINE; LIPOSOME; N' CHOLESTERYLOXYCARBONYL 3,7 DIAZANONANE 1,9 DIAMINE; NUCLEIC ACID; PLASMID DNA; POLYAMINE DERIVATIVE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; CELL CULTURE; CONCENTRATION (PARAMETERS); CONTROLLED STUDY; CYTOTOXICITY; DOWN REGULATION; GENE EXPRESSION; GENE FUNCTION; GENE SILENCING; GENE TARGETING; GENOMICS; HUMAN; HUMAN CELL; MAMMAL CELL; NONHUMAN; OPTIMAL DRUG DOSE; PARAMETER; PARTICLE SIZE; PRIORITY JOURNAL; PROTEIN CONTENT; PROTEIN EXPRESSION; PROTEIN FUNCTION; RNA INTERFERENCE; RNA SEQUENCE;

ANIMALIA; MAMMALIA;

EID: 6344291687     PISSN: 00062960     EISSN: None     Source Type: Journal    
DOI: 10.1021/bi048950a     Document Type: Article

References (43)

1. Novina, C. D., and Sharp, P. A. (2004) The RNAi revolution, Nature 430, 161-164.
2. Tang, G. L., Reinhart, B. J., Bartel, D. P., and Zamore, P. D. (2003) A biochemical framework for RNA silencing in plants, Genes Dev. 17, 49-63.
3. Catalanotto, C., Azzalin, G., Macino, G., and Cogoni, C. (2002) Involvement of small RNAs and role of the qde genes in the gene silencing pathway in Neurospora, Genes Dev. 16, 790-795.
4. Wassenegger, M., Heimes, S., Riedel, L., and Sanger, H. L. (1994) RNA-Directed De-Novo Methylation of Genomic Sequence in Plants, Cell 76, 567-576.
5. Hamilton, A. J., and Baulcombs, D. C. (1999) A species of small antisense RNA in posttranscriptional gene silencing in plants, Science 286, 950-952.
6. Fire, A., Xu, S. Q., Montgomery, M. K., Kostas, S. A., Driver, S. E., et al. (1998) Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans, Nature 391, 806-811.
7. Hutvagner, G., McLachlan, J., Pasquinelli, A. E., Balint, E., Tuschl, T., et al. (2001) A cellular function for the RNA-interference enzyme Dicer in the maturation of the let-7 small temporal RNA, Science 293, 834-838.
8. Bernstein, E., Caudy, A. A., Hammond, S. M., and Hannon, G. J. (2001) Role for a bidentate ribonuclease in the initiation step of RNA interference, Nature 409, 363-366.
9. Hammond, S. M., Boettcher, S., Caudy, A. A., Kobayashi, R., and Hannon, G. J. (2001) Argonaute2, a link between genetic and biochemical analysis of RNAi, Science 293, 1146-1150.
10. Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., et al. (2001) Duplexes of 21-nucleotide RNAs mediate RNA intereference in cultured mammalian cells, Nature 411, 494-498.
11. Hasuwa, H., Kaseda, K., Einarsdottir, T., and Okabe, M. (2002) Small Interfering RNA and Gene Silencing in Transgenic Mice and Rats, FEBS Lett. 532, 227-230.
12. Sorensen, D. R., Leirdal, M., and Sioud, M. (2003) Gene Silencing by Systemic Delivery of Synthetic siRNAs in Adult Mice, J. Mol. Biol. 327, 761-766.
13. Sledz, C. A., Holko, M., de Veer, M. J., Silverman, R. H., and Williams, B. R. G. (2003) Activation of the Interferon System by Short-Interfering RNAs, Nat. Cell Biol. 5, 834-839.
14. Bridge, A. J., Pebernard, S., Ducraux, A., Nicoulaz, A. L., and Iggo, R. (2003) Induction of an Interferon Response by RNAi Vectors in Mammalian Cells, Nat. Genet. 34, 263-264.
15. Felgner, P. L., Gadek, T. R., Holm, M., Roman, R., Chan, H. W., et al. (1987) Lipofection: A Highly Efficient, Lipid-Mediated DNA-Transfection Procedure, Proc. Natl. Acad. Sci. U.S.A. 84, 7413-7417.
16. Lasic, D. D., and Papahadjopoulos, D. (1995) Liposomes Revisited, Science 267, 1275-1276.
17. Zelphati, O., Nguyen, C., Ferrari, M., Feigner, J., Tsai, Y., et al. (1998) Stable and Monodisperse Lipoplex Formulations for Gene Delivery, Gene Ther. 5, 1272-1282.
18. Hansma, H. G., Golan, R., Hsieh, W., Lollo, C. P., MullenLey, P., et al. (1998) DNA Condensation for Gene Therapy as Monitored by Atomic Force Microscopy, Nucleic Acids Res. 26, 2481-2487.
19. Golan, R., Pietrasanta, L. I., Hsieh, W., and Hansma, H. G. (1999) DNA Toroids: Stages in Condensation, Biochemistry 38, 14069-14076.
20. Keller, M., Harbottle, R. P., Perouzel, E., Morvane, C., Imran, S., et al. (2003) Nuclear Localization Sequence (NLS)-Templated Non-Viral Gene Delivery Vectors: Investigation of Intracellular Trafficking Events of LMD and LD Vector Systems, ChemBioChem 4, 286-298.
21. Bloomfield, V. A. (1996) DNA Condensation, Curr. Opin. Struct. Biol. 6, 334-341.
22. Boulikas, T., and Martin, F. (1997) Histones, Protamine, and Polylysine but not Poly(E:K) Enhance Transfection Efficiency, Int. J. Oncol. 10, 317-322.
23. Stewart, L., Manvell, M., Hillery, E., Etheridge, C. J., Cooper, R. G., et al. (2001) Cationic Lipids for Gene Therapy Part 4: Physicochemical Analysis of Cationic Liposome-DNA Complexes (Lipoplexes) with Respect to in Vitro and in Vivo Gene Delivery Efficiency, J. Chem. Soc., Perkin Trans. 2, 624-632.
24. Keller, M., Tagawa, T., Preuss, M., and Miller, A. D. (2002) Biophysical Characterization of the DNA Binding and Condensing Properties of Adenoviral Core Peptide μ (mu), Biochemistry 41, 652-659.
25. Bloomfield, V. A. (1991) Condensation of DNA by Multivalent Cations: Considerations on Mechanism, Biopolymers 31, 1471-1481.
26. Kreiss, P., Cameron, B., Rangara, R., Mailhe, P., Aguerre-Charriol, O., et al. (1999) Plasmid DNA Size does not Affect the Physicochemical Properties of Lipoplexes but Modulates Gene Transfer Efficiency, Nucleic Acids Res. 27, 3792-3798.
27. Chesnoy, S., and Huang, L. (1999) DNA Condensed by Polycations and Lipids for Gene Transfer, S.T.P. Pharma Sci. 9, 5-12.
28. Ross, P. C., and Hui, S. W. (1999) Lipoplex Size is a Major Determinant of In Vitro Lipofection Efficiency, Gene Ther. 6, 651-659.
29. Simberg, D., Danino, D., Talmon, Y., Minsky, A., Ferrari, M. E., et al. (2001) Phase behavior, DNA ordering, and size instability of cationic lipoplexes: Relevance to optimal transfection activity, J. Biol. Chem. 276, 47453-47459.
30. Zuidam, N. J., HirschLerner, D., Margulies, S., and Barenholz, Y. (1999) Lamellarity of cationic liposomes and mode of preparation of lipoplexes affect transfection efficiency, Biochim. Biophys. Acta 1419, 207-220.
31. Zuidam, N. J., and Barenholz, Y. (1999) Characterization of DNA-lipid complexes commonly used for gene delivery, Int. J. Pharm. 183, 43-46.
32. Keller, M., Jorgensen, M. R., Perouzel, E., and Miller, A. D. (2003) Thermodynamic Aspects and Biological Profile of CDAN/DOPE and DC-Chol/DOPE Lipoplexes, Biochemistry 42, 6067-6077.
33. Shi, F. X., Wasungu, L., Nomden, A., Stuart, M. C. A., Polushkin, E., et al. (2002) Interference of Poly(ethylene glycol)-Lipid Analogues With Cationic-Lipid-Mediated Delivery of Oligonucleotides; Role of Lipid Exchangeability and Non-Lamellar Transitions, Biochem. J. 366, 333-341.
34. + channel function in oocyte expression studies, J. Biol. Chem. 278, 11739-11745.
35. Semizarov, D., Frost, L., Sarthy, A., Kroeger, P., Halbert, D. N., et al. (2003) Specificity of Short Interfering RNA Determined Through Gene Expression Signatures, Proc. Natl. Acad. Sci. U.S.A. 100, 6347-6352.
36. Chi, J. T., Chang, H. Y., Wang, N. N., Chang, D. S., Dunphy, N., et al. (2003) Genomewide View of Gene Silencing by Small Interfering RNAs, Proc. Natl. Acad. Sci. U.S.A. 100, 6343-6346.
37. Fisher, A. A., Ye, D. J., Sergueev, D. S., Fisher, M. H., Shaw, B. R., et al. (2002) Evaluating the Specificity of Antisense Oligonucleotide Conjugates: A DNA Array Analysis, J. Biol. Chem. 277, 22980-22984.
38. Cho, Y. S., Kim, M. K., Cheadle, C., Neary, C., Becker, K. G., et al. (2001) Antisense DNAs as Multisite Genomic Modulators Identified by DNA Microarray, Proc. Natl. Acad. Sci. U.S.A. 98, 9819-9823.
39. Stein, C. A. (1995) Does Antisense Exist, Nat. Med. 1, 1119-1121.
40. Brukner, I., and Tremblay, G. A. (2000) Cellular Proteins Prevent Antisense Phosphorothioate Oligonucleotide (SdT18) to Target Sense RNA (rA18): Development of a New in Vitro Assay, Biochemistry 39, 11463-11466.
41. Ui-Tei, K., Naito, Y., Takahashi, F., Haraguchi, T., Ohki-Hamazaki, H., et al. (2004) Guidelines for the selection of highly effective siRNA sequences for mammalian and chick RNA interference, Nucleic Acids Res. 32, 936-948.
42. Nakanishi, M., Mizuguchia, H., Ashihara, K., Senda, T., Akuta, T., et al. (1998) Gene transfer vectors based on Sendai virus, J. Controlled Release 54, 61-68.
43. Colin, M., Harbottle, R. P., Knight, A., Kornprobst, M., Cooper, R. G., et al. (1998) Liposomes Enhance Delivery and Expression of an RGD-oligolysine Gene Transfer Vector in Human Tracheal Cells, Gene Ther. 5, 1488-1498.