Systemic delivery of small RNA using lipid nanoparticles

Biological and Pharmaceutical Bulletin

Volumn 37, Issue 2, 2014, Pages 201-205

  Asai, Tomohiro ^a   Oku, Naoto ^a  

^a UNIVERSITY OF SHIZUOKA   (Japan)

Author keywords

Lipid nanoparticle; MicroRNA; RNA interference; Small interfering RNA; Systemic delivery

Indexed keywords

ANIMALS; DRUG DELIVERY SYSTEMS; HUMANS; LIPIDS; MICRORNAS; NANOPARTICLES; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 84893228643     PISSN: 09186158     EISSN: 13475215     Source Type: Journal    
DOI: 10.1248/bpb.13-00744     Document Type: Review

References (30)

1. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature, 391, 806-811 (1998).
2. Bernstein E, Caudy AA, Hammond SM, Hannon GJ. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature, 409, 363-366 (2001).
3. Liu J, Carmell MA, Rivas FV, Marsden CG, Thomson JM, Song JJ, Hammond SM, Joshua-Tor L, Hannon GJ. Argonaute2 is the catalytic engine of mammalian RNAi. Science, 305, 1437-1441 (2004).
4. Hutvágner G, Zamore PD. A microRNA in a multiple-turnover RNAi enzyme complex. Science, 297, 2056-2060 (2002).
5. Haley B, Zamore PD. Kinetic analysis of the RNAi enzyme complex. Nat. Struct. Mol. Biol., 11, 599-606 (2004).
6. Resnier P, Montier T, Mathieu V, Benoit JP, Passirani C. A review of the current status of siRNA nanomedicines in the treatment of cancer. Biomaterials, 34, 6429-6443 (2013).
7. Strumberg D, Schultheis B, Traugott U, Vank C, Santel A, Keil O, Giese K, Kaufmann J, Drevs J. Drevs, J. Phase I clinical development of Atu027, a siRNA formulation targeting PKN3 in patients with advanced solid tumors. Int. J. Clin. Pharmacol. Ther., 50, 76-78 (2012).
8. Davis ME, Zuckerman JE, Choi CH, Seligson D, Tolcher A, Alabi CA, Yen Y, Heidel JD, Ribas A. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature, 464, 1067-1070 (2010).
9. Matsumura Y, Maeda H. A new concept for macromolecular therapeutics in cancer chemotherapy: mechanism of tumoritropic accumulation of proteins and the antitumor agent smancs. Cancer Res., 46, 6387-6392 (1986).
10. Valadi H, Ekstrom K, Bossios A, Sjostrand M, Lee JJ, Lotvall JO. Exosome-mediated transfer of mRNAs and microRNAs is a novel mechanism of genetic exchange between cells. Nat. Cell Biol., 9, 654-659 (2007).
11. Zimmermann TS, Lee AC, Akinc A, Bramlage B, Bumcrot D, Fedoruk MN, Harborth J, Heyes JA, Jeffs LB, John M, Judge AD, Lam K, McClintock K, Nechev LV, Palmer LR, Racie T, Rohl I, Seiffert S, Shanmugam S, Sood V, Soutschek J, Toudjarska I, Wheat AJ, Yaworski E, Zedalis W, Koteliansky V, Manoharan M, Vornlocher HP, MacLachlan I. RNAi-mediated gene silencing in non-human primates. Nature, 441, 111-114 (2006).
12. Semple SC, Akinc A, Chen J, Sandhu AP, Mui BL, Cho CK, Sah DW, Stebbing D, Crosley EJ, Yaworski E, Hafez IM, Dorkin JR, Qin J, Lam K, Rajeev KG, Wong KF, Jeffs LB, Nechev L, Eisenhardt ML, Jayaraman M, Kazem M, Maier MA, Srinivasulu M, Weinstein MJ, Chen Q, Alvarez R, Barros SA, De S, Klimuk SK, Borland T, Kosovrasti V, Cantley WL, Tam YK, Manoharan M, Ciufolini MA, Tracy MA, de Fougerolles A, MacLachlan I, Cullis PR, Madden TD, Hope MJ. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol., 28, 172-176 (2010).
13. Barros SA, Gollob JA. Safety profile of RNAi nanomedicines. Adv. Drug Deliv. Rev., 64, 1730-1737 (2012).
14. Santel A, Aleku M, Roder N, Mopert K, Durieux B, Janke O, Keil O, Endruschat J, Dames S, Lange C, Eisermann M, Loffler K, Fechtner M, Fisch G, Vank C, Schaeper U, Giese K, Kaufmann J. Atu027 prevents pulmonary metastasis in experimental and spontaneous mouse metastasis models. Clin. Cancer Res., 16, 5469-5480 (2010).
15. Aleku M, Schulz P, Keil O, Santel A, Schaeper U, Dieckhoff B, Janke O, Endruschat J, Durieux B, Roder N, Loffler K, Lange C, Fechtner M, Mopert K, Fisch G, Dames S, Arnold W, Jochims K, Giese K, Wiedenmann B, Scholz A, Kaufmann J. Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression. Cancer Res., 68, 9788-9798 (2008).
16. Kawai T, Akira S. Antiviral signaling through pattern recognition receptors. J. Biochem., 141, 137-145 (2007).
17. Judge AD, Sood V, Shaw JR, Fang D, McClintock K, MacLachlan I. Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA. Nat. Biotechnol., 23, 457-462 (2005).
18. Judge AD, Bola G, Lee AC, MacLachlan I. Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo. Mol. Ther., 13, 494-505 (2006).
19. Robbins M, Judge A, Liang L, McClintock K, Yaworski E, Mac Lachlan I. 2′ -O-Methyl-modified RNAs act as TLR7 antagonists. Mol. Ther., 15, 1663-1669 (2007).
20. Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Rohl I, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature, 432, 173-178 (2004).
21. Wolfrum C, Shi S, Jayaprakash KN, Jayaraman M, Wang G, Pandey RK, Rajeev KG, Nakayama T, Charrise K, Ndungo EM, Zimmermann T, Koteliansky V, Manoharan M, Stoffel M. Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat. Biotechnol., 25, 1149-1157 (2007).
22. Asai T, Matsushita S, Kenjo E, Tsuzuku T, Yonenaga N, Koide H, Hatanaka K, Dewa T, Nango M, Maeda N, Kikuchi H, Oku N. Dicetyl phosphate- tetraethylenepentamine-based liposomes for systemic siRNA delivery. Bioconjug. Chem., 22, 429-435 (2011).
23. Yonenaga N, Kenjo E, Asai T, Tsuruta A, Shimizu K, Dewa T, Nango M, Oku N. RGD-based active targeting of novel polycation liposomes bearing siRNA for cancer treatment. J. Control. Release, 160, 177-181 (2012).
24. Kenjo E, Asai T, Yonenaga N, Ando H, Ishii T, Hatanaka K, Shimizu K, Urita Y, Dewa T, Nango M, Tsukada H, Oku N. Systemic delivery of small interfering RNA by use of targeted polycation liposomes for cancer therapy. Biol. Pharm. Bull., 36, 287-291 (2013).
25. Vlassov AV, Magdaleno S, Setterquist R, Conrad R. Exosomes: current knowledge of their composition, biological functions, and diagnostic and therapeutic potentials. Biochim. Biophys. Acta, 1820, 940-948 (2012).
26. Subra C, Laulagnier K, Perret B, Record M. Exosome lipidomics unravels lipid sorting at the level of multivesicular bodies. Biochimie, 89, 205-212 (2007).
27. Li SD, Huang L. Nanoparticles evading the reticuloendothelial system: role of the supported bilayer. Biochim. Biophys. Acta, 1788, 2259-2266 (2009).
28. Kooijmans SA, Vader P, van Dommelen SM, van Solinge WW, Schiffelers RM. Exosome mimetics: a novel class of drug delivery systems. Int. J. Nanomedicine, 7, 1525-1541 (2012).
29. Alvarez-Erviti L, Seow Y, Yin H, Betts C, Lakhal S, Wood MJ. Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat. Biotechnol., 29, 341-345 (2011).
30. Wahlgren J, De L Karlson T, Brisslert M, Vaziri Sani F, Telemo E, Sunnerhagen P, Valadi H. Plasma exosomes can deliver exogenous short interfering RNA to monocytes and lymphocytes. Nucleic Acids Res., 40, e130 (2012).