Mechanism of macromolecular structure evolution in self-assembled lipid nanoparticles for siRNA delivery

Langmuir

Volumn 30, Issue 16, 2014, Pages 4613-4622

  Gindy, Marian E ^a   Difelice, Katherine ^a   Kumar, Varun ^a,b   Prud'Homme, Robert K ^b   Celano, Robert ^a   Haas, R Matthew ^a   Smith, Jeffrey S ^a   Boardman, David ^a  

^a MERCK RESEARCH LABORATORIES   (United States)

^b Princeton University   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

NANOPARTICLES; PRECIPITATION (CHEMICAL); RNA; SELF ASSEMBLY;

ANTI-SOLVENT PRECIPITATIONS; MACROMOLECULAR STRUCTURES; PRECIPITATION PROCESS; SMALL ANGLE X-RAY SCATTERING; SMALL INTERFERING RNA; STRUCTURAL REORGANIZATION; STRUCTURE-ACTIVITY RELATIONSHIPS; WELL-ORDERED STRUCTURE;

MACROMOLECULES;

LIPID; NANOPARTICLE; SMALL INTERFERING RNA;

CHEMISTRY; STRUCTURE ACTIVITY RELATION;

LIPIDS; NANOPARTICLES; RNA, SMALL INTERFERING; STRUCTURE-ACTIVITY RELATIONSHIP;

EID: 84899630282     PISSN: 07437463     EISSN: 15205827     Source Type: Journal    
DOI: 10.1021/la500630h     Document Type: Article

References (33)

1. Fire, A.; Xu, S. Q.; 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 1998, 391 (6669) 806-811
2. 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 2001, 411 (6836) 494-498
3. Zimmermann, T. S.; Lee, A. C. H.; Akinc, A.; Bramlage, B.; Bumcrot, D.; Fedoruk, M. N.; Harborth, J.; Heyes, J. A.; Jeffs, L. B.; John, M.; Judge, A. D.; Lam, K.; McClintock, K.; Nechev, L. V.; Palmer, L. R.; Racie, T.; Rohl, I.; Seiffert, S.; Shanmugam, S.; Sood, V.; Soutschek, J.; Toudjarska, I.; Wheat, A. J.; Yaworski, E.; Zedalis, W.; Koteliansky, V.; Manoharan, M.; Vornlocher, H. P.; MacLachlan, I. RNAi-mediated gene silencing in non-human primates Nature 2006, 441 (7089) 111-114
4. Davis, M. E.; Zuckerman, J. E.; Choi, C. H. J.; Seligson, D.; Tolcher, A.; Alabi, C. A.; Yen, Y.; Heidel, J. D.; Ribas, A. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles Nature 2010, 464 (7291) 1067-1070
5. Whitehead, K. A.; Langer, R.; Anderson, D. G. Knocking down barriers: advances in siRNA delivery Nat. Rev. Drug Discovery 2009, 8 (2) 129-138
6. Mintzer, M. A.; Simanek, E. E. Nonviral vectors for gene delivery Chem. Rev. 2009, 109 (2) 259-302
7. Davidson, B. L.; McCray, P. B. Current prospects for RNA interference-based therapies Nat. Rev. Genet. 2011, 12 (5) 329-340
8. Tseng, Y. C.; Mozumdar, S.; Huang, L. Lipid-based systemic delivery of siRNA Adv. Drug Delivery Rev. 2009, 61 (9) 721-731
9. Li, W.; Szoka, F. C., Jr. Lipid-based nanoparticles for nucleic acid delivery Pharm. Res. 2007, 24 (3) 438-449
10. Wasungu, L.; Hoekstra, D. Cationic lipids, lipoplexes and intracellular delivery of genes J. Controlled Release 2006, 116 (2) 255-264
11. Senior, J.; Gregoriadis, G. Stability of small unilamellar liposomes in serum and clearance from the circulation-the effect of the phospholipid and cholesterol components Life Sci. 1982, 30 (24) 2123-2136
12. Klibanov, A. L.; Maruyama, K.; Torchilin, V. P.; Huang, L. Amphipathic polyethyleneglycols effectively prolong the circulation time of liposomes FEBS Lett. 1990, 268 (1) 235-237
13. Akinc, A.; Querbes, W.; De, S. M.; Qin, J.; Frank-Kamenetsky, M.; Jayaprakash, K. N.; Jayaraman, M.; Rajeev, K. G.; Cantley, W. L.; Dorkin, J. R.; Butler, J. S.; Qin, L. L.; Racie, T.; Sprague, A.; Fava, E.; Zeigerer, A.; Hope, M. J.; Zerial, M.; Sah, D. W. Y.; Fitzgerald, K.; Tracy, M. A.; Manoharan, M.; Koteliansky, V.; de Fougerolles, A.; Maier, M. A. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms Mol. Ther. 2010, 18 (7) 1357-1364
14. Semple, S. C.; Akinc, A.; Chen, J.; Sandhu, A. P.; Mui, B. L.; Cho, C. K.; Sah, D. W. Y.; Stebbing, D.; Crosley, E. J.; Yaworski, E.; Hafez, I. M.; Dorkin, J. R.; Qin, J.; Lam, K.; Rajeev, K. G.; Wong, K. F.; Jeffs, L. B.; Nechev, L.; Eisenhardt, M. L.; Jayaraman, M.; Kazem, M.; Maier, M. A.; Srinivasulu, M.; Weinstein, M. J.; Chen, Q.; Alvarez, R.; Barros, S. A.; De, S.; Klimuk, S. K.; Borland, T.; Kosovrasti, V.; Cantley, W. L.; Tam, Y. K.; Manoharan, M.; Ciufolini, M. A.; Tracy, M. A.; de Fougerolles, A.; MacLachlan, I.; Cullis, P. R.; Madden, T. D.; Hope, M. J. Rational design of cationic lipids for siRNA delivery Nat. Biotechnol. 2010, 28 (2) 172-176
15. Stanton, M. G.; Budzik, B. W.; Beutner, B. W.; Liao, H. Novel Low Molecular Weight Cationic Lipids for Oligonucleotide Delivery, 2012.
16. Ewert, K.; Slack, N. L.; Ahmad, A.; Evans, H. M.; Lin, A. J.; Samuel, C. E.; Safinya, C. R. Cationic lipid-DNA complexes for gene therapy: Understanding the relationship between complex structure and gene delivery pathways at the molecular level Curr. Med. Chem. 2004, 11 (2) 133-149
17. Hoekstra, D.; Rejman, J.; Wasungu, L.; Shi, F.; Zuhorn, I. Gene delivery by cationic lipids: in and out of an endosome Biochem. Soc. Trans. 2007, 35, 68-71
18. Leal, C.; Bouxsein, N. F.; Ewert, K. K.; Safinya, C. R. Highly efficient gene silencing activity of siRNA embedded in a nanostructured gyroid cubic lipid matrix J. Am. Chem. Soc. 2010, 132 (47) 16841-16847
19. Gindy, M. E.; Leone, A. M.; Cunningham, J. J. Challenges in the pharmaceutical development of lipid-based short interfering ribonucleic acid therapeutics Expert Opin. Drug Delivery 2012, 9 (2) 171-182
20. Maurer, N.; Wong, K. F.; Stark, H.; Louie, L.; McIntosh, D.; Wong, T.; Scherrer, P.; Semple, S. C.; Cullis, P. R. Spontaneous entrapment of polynucleotides upon electrostatic interaction with ethanol-destabilized cationic liposomes Biophys. J. 2001, 80 (5) 2310-2326
21. Jeffs, L. B.; Palmer, L. R.; Ambegia, E. G.; Giesbrecht, C.; Ewanick, S.; MacLachlan, I. A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA Pharm. Res. 2005, 22 (3) 362-372
22. Jahn, A.; Stavis, S. M.; Hong, J. S.; Vreeland, W. N.; Devoe, D. L.; Gaitan, M. Microfluidic mixing and the formation of nanoscale lipid vesicles ACS Nano 2010, 4 (4) 2077-2087
23. Abrams, M. T.; Koser, M. L.; Seitzer, J.; Williams, S. C.; DiPietro, M. A.; Wang, W.; Shaw, A. W.; Mao, X.; Jadhav, V.; Davide, J. P.; Burke, P. A.; Sachs, A. B.; Stirdivant, S. M.; Sepp-Lorenzino, L. Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: Effect of dexamethasone co-treatment Mol. Therapy 2010, 18 (1) 171-180
24. Tao, W.; Davide, J. P.; Cai, M.; Zhang, G.-J.; South, V. J.; Matter, A.; Ng, B.; Zhang, Y.; Sepp-Lorenzino, L., Noninvasive imaging of lipid nanoparticle-mediated systemic delivery of small-interfering RNA to the liver. Mol. Therapy 2010, 18, (9), 1657-1666.
25. Gindy, M. E.; Panagiotopoulos, A. Z.; Prud'homme, R. K. Composite block copolymer stabilized nanoparticles: Simultaneous encapsulation of organic actives and inorganic nanostructures Langmuir 2008, 24 (1) 83-90
26. Ly, H. V.; Longo, M. L. The influence of short-chain alcohols on interfacial tension, mechanical properties, area/molecule, and permeability of fluid lipid bilayers Biophys. J. 2004, 87 (2) 1013-1033
27. Patra, M.; Salonen, E.; Terama, E.; Vattulainen, I.; Faller, R.; Lee, B. W.; Holopainen, J.; Karttunen, M. Under the influence of alcohol: The effect of ethanol and methanol on lipid bilayers Biophys. J. 2006, 90 (4) 1121-1135
28. Torchilin, V. P. Micellar nanocarriers: Pharmaceutical perspectives Pharm. Res. 2007, 24 (1) 1-16
29. Vonarbourg, A.; Passirani, C.; Saulnier, P.; Simard, P.; Leroux, J. C.; Benoit, J. P. Evaluation of pegylated lipid nanocapsules versus complement system activation and macrophage uptake J. Biomed. Mater. Res., Part A 2006, 78A (3) 620-628
30. Iden, D. L.; Allen, T. M. In vitro and in vivo comparison of immunoliposomes made by conventional coupling techniques with those made by a new post-insertion approach Biochim. Biophys. Acta, Biomembr. 2001, 1513 (2) 207-216
31. Ashok, B.; Arleth, L.; Hjelm, R. P.; Rubinstein, I.; Onyuksel, H. In vitro characterization of PEGylated phospholipid micelles for improved drug solubilization: Effects of PEG chain length and PC incorporation J. Pharm. Sci. 2004, 93 (10) 2476-2487
32. Hao, J.; Yuan, G.; He, W.; Cheng, H.; Han, C. C.; Wu, C. Interchain Hydrogen-Bonding-Induced Association of Poly(acrylic acid)-graft-poly(ethylene oxide) in Water Macromolecules 2010, 43 (4) 2002-2008
33. Lv, H.; Zhang, S.; Wang, B.; Cui, S.; Yan, J. Toxicity of cationic lipids and cationic polymers in gene delivery J. Controlled Release 2006, 114 (1) 100-109