Cell-penetrating cationic siRNA and lipophilic derivatives efficient at nanomolar concentrations in the presence of serum and albumin

Journal of Controlled Release

Volumn 170, Issue 1, 2013, Pages 92-98

  Perche, Phanélie ^a   Nothisen, Marc ^a   Bagilet, Jérémy ^a   Behr, Jean Paul ^a   Kotera, Mitsuharu ^a   Remy, Jean Serge ^a  

^a UNIVERSITÉ DE STRASBOURG   (France)

Author keywords

Cationic oligonucleotides; Chemically modified siRNA; Cholesteryl siRNA; Lipophilic siRNA; RNA interference; ZNA

Indexed keywords

BODY FLUIDS; OLIGONUCLEOTIDES; RNA;

CHEMICALLY MODIFIED; CHOLESTERYL-SIRNA; GENE SILENCING; IN-VITRO EVALUATION; LIPOPHILIC SIRNA; NANOMOLAR CONCENTRATION; ORDERS OF MAGNITUDE; RNA INTERFERENCE;

GENE THERAPY;

ALBUMIN; CATION; CELL PENETRATING PEPTIDE; CHOLESTERYL SMALL INTERFERING RNA; LIPID; LUCIFERASE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ARTICLE; CONCENTRATION (PARAMETERS); CONTROLLED STUDY; DRUG DEGRADATION; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG SCREENING; DRUG SYNTHESIS; GENE SILENCING; HUMAN; HUMAN CELL; IN VITRO STUDY; PRIORITY JOURNAL;

EID: 84878740017     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2013.04.013     Document Type: Article

References (23)

1. A. Fire, S. Xu, M.K. Montgomery, S.A. Kostas, S.E. Driver, C.C. Mello, Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans, Nature 391 (1998) 806-811. (Pubitemid 28099681)
2. S.M. Elbashir, J. Harborth, W. Lendeckel, A. Yalcin, K. Weber, T. Tuschl, Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells, Nature 411 (2001) 494-498. (Pubitemid 32494397)
3. J. Kurreck, RNA interference: from basic research to therapeutic applications, Angew. Chem. Int. Ed. 48 (2009) 1378-1398.
4. A.D. Judge, V. Sood, J.R. Shaw, D. Fang, K. McClintock, I. MacLachlan, Sequencedependent stimulation of the mammalian innate immune response by synthetic siRNA, Nat. Biotechnol. 23 (2005) 457-462. (Pubitemid 41724921)
5. Z. Ma, J. Li, F. He, A. Wilson, B. Pitt, S. Li, Cationic lipids enhance siRNA-mediated interferon response in mice, Biochem. Biophys. Res. Commun. 330 (2005) 755-759. (Pubitemid 40462151)
6. M.Meyer,C.Dohmen, A.Philipp,D.Kiener, G.Maiwald, C. Scheu, M. Ogris, E.Wagner, Synthesis and biological evaluation of a bioresponsive and endosomolytic siRNA- polymer conjugate, Mol. Pharm. 6 (2009) 752-762.
7. S.A.Moschos, S.W. Jones, M.M. Perry, A.E. Williams, J.S. Erjefalt, J.J. Turner, P.J. Barnes, B.S. Sproat, M.J. Gait, M.A. Lindsay, Lung delivery studies using siRNA conjugated to TAT(48-60) and penetratin reveal peptide induced reduction in gene expression and induction of innate immunity, Bioconjug. Chem. 18 (2007) 1450-1459. (Pubitemid 47464052)
8. A. Muratovska, M.R. Eccles, Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells, FEBS Lett. 558 (2004) 63-68. (Pubitemid 38167411)
9. J. Soutschek, A. Akinc, B. Bramlage, K. Charisse, R. Constien, M. Donoghue, S. Elbashir, A. Geick, P. Hadwiger, J. Harborth, M. John, V. Kesavan, G. Lavine, R.K. Pandey, T. Racie, K.G. Rajeev, I. Rohl, I. Toudjarska, G. Wang, S. Wuschko, D. Bumcrot, V. Koteliansky, S. Limmer, M. Manoharan, H.-P. Vornlocher, Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs, Nature 432 (2004) 173-178. (Pubitemid 39545841)
10. C. Wolfrum, S. Shi, K.N. Jayaprakash, M. Jayaraman, G. Wang, R.K. Pandey, K.G. Rajeev, T. Nakayama, K. Charrise, E.M. Ndungo, T. Zimmermann, V. Koteliansky, M. Manoharan, M. Stoffel, Mechanisms and optimization of in vivo delivery of lipophilic siRNAs, Nat. Biotechnol. 25 (2007) 1149-1157. (Pubitemid 47538115)
11. A. Eguchi, S.F. Dowdy, siRNA delivery using peptide transduction domains, Trends Pharmacol. Sci. 30 (2009) 341-345.
12. http://www.dharmacon.com/uploadedFiles/Home/Resources/Protocols/ accelldelivery- protocol.pdf.
13. E. Voirin, J.-P. Behr, M. Kotera, Versatile synthesis of oligodeoxyribonucleotide- oligospermine conjugates, Nat. Protoc. 2 (2007) 1360-1367. (Pubitemid 46952318)
14. B. Pons,M. Kotera, G. Zuber, J.-P. Behr, Online synthesis of diblock cationic oligonucleotides for enhanced hybridization to their complementary sequence, ChemBioChem7 (2006) 1173-1176. (Pubitemid 44230839)
15. M. Nothisen, M. Kotera, E. Voirin, J.-S. Remy, J.-P. Behr, Cationic siRNAs provide carrier-free gene silencing in animal cells, J. Am. Chem. Soc. 131 (2009) 17730-17731.
16. http://www.glenresearch.com/GlenReports/GR19-2.pdf, (in).
17. E. Morin, M. Nothisen, A. Wagner, J.-S. Remy, Cationic polydiacetylene micelles for gene delivery, Bioconjug. Chem. 22 (2011) 1916-1923.
18. J. Martinez, A. Patkaniowska, H. Urlaub, R. Lührmann, T. Tuschl, Single-stranded antisense siRNAs guide target RNA cleavage in RNAi, Cell 110 (2002) 563-574. (Pubitemid 35247837)
19. S.M. Elbashir, W. Lendeckel, T. Tuschl, RNA interference is mediated by 21- and 22-nucleotide RNAs, Genes Dev. 15 (2001) 188-200. (Pubitemid 32107664)
20. A. Khvorova, A. Reynolds, S.D. Jayasena, Functional siRNAs and miRNAs exhibit strand bias, Cell 115 (2003) 209-216. (Pubitemid 37329584)
21. D.S. Schwarz, G. Hutvagner, T. Du, Z. Xu, N. Aronin, P.D. Zamore, Asymmetry in the assembly of the RNAi enzyme complex, Cell 115 (2003) 199-208. (Pubitemid 37329583)
22. S. Shukla, C. Sumaria, P. Pradeepkumar, Exploring chemical modifications for siRNA therapeutics: a structural and functional outlook, ChemMedChem 5 (2010) 328-349.
23. S. Lau, B. Graham, N. Cao, B.J. Boyd, C.W. Pouton, P.J.White, Enhanced extravasation, stability and in vivo cardiac gene silencing via in situ siRNA-albumin conjugation, Mol. Pharm. 9 (2012) 71-80.