Quantitation of physiological and biochemical barriers to siRNA liver delivery via lipid nanoparticle platform

Molecular Pharmaceutics

Volumn 11, Issue 5, 2014, Pages 1424-1434

  Xu, Yan ^a   Ou, Mei ^a   Keough, Ed ^a   Roberts, Jeff ^a,b   Koeplinger, Ken ^a   Lyman, Mike ^a   Fauty, Scott ^a   Carlini, Ed ^a   Stern, Melissa ^a   Zhang, Rena ^a   Yeh, Suzie ^a   Mahan, Elizabeth ^a   Wang, Yi ^a   Slaughter, Don ^a,c   Gindy, Marian ^a   Raab, Conrad ^a   Thompson, Charles ^a   Hochman, Jerome ^a  

^a MERCK RESEARCH LABORATORIES   (United States)

^b GLAXOSMITHKLINE   (United States)

^c Montgomery County Community College   (United States)

Author keywords

delivery efficiency; lipid nanoparticle; liver; siRNA; subcellular fractionation

Indexed keywords

ARGONAUTE 2 PROTEIN; LIPID; LIPID A; LIPID NANOPARTICLE; MACROGOL; MESSENGER RNA; NANOPARTICLE; NUCLEOTIDE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL FRACTIONATION; CELL ISOLATION; CELL SURFACE; CELL VACUOLE; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG DISTRIBUTION; DRUG FORMULATION; DRUG LIVER LEVEL; DRUG PENETRATION; DRUG POTENCY; DRUG UPTAKE; ENDOSOME; FEMALE; GENE SILENCING; INTERNALIZATION; LIVER CELL; LIVER PERFUSION; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PLASMA; PRIORITY JOURNAL; QUANTITATIVE ANALYSIS; RAT; RNA BINDING; RNA INTERFERENCE; ANIMAL; CELL CULTURE; CHEMISTRY; FLUORESCENCE MICROSCOPY; LIVER; METABOLISM; SPRAGUE DAWLEY RAT;

ANIMALS; CELLS, CULTURED; FEMALE; HEPATOCYTES; LIPIDS; LIVER; MICROSCOPY, FLUORESCENCE; NANOPARTICLES; RATS; RATS, SPRAGUE-DAWLEY; RNA, SMALL INTERFERING;

EID: 84899843329     PISSN: 15438384     EISSN: 15438392     Source Type: Journal    
DOI: 10.1021/mp400584h     Document Type: Article

References (30)

1. Burnett, J. C.; Rossi, J. J.; Tiemann, K. Current progress of siRNA/shRNA therapeutics in clinical trials Biotechnol. J. 2011, 6 (9) 1130-46
2. Schmitz, J. C.; Chen, T. M.; Chu, E. Small interfering double-stranded RNAs as therapeutic molecules to restore chemosensitivity to thymidylate synthase inhibitor compounds Cancer Res. 2004, 64 (4) 1431-5
3. Foster, D. J. Comprehensive evaluation of canonical versus Dicer-substrate siRNA in vitro and in vivo RNA 2012, 18 (3) 557-68
4. Abrams, M. T. Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: effect of dexamethasone co-treatment Mol. Ther. 2010, 18 (1) 171-80
5. Wei, J. RNA-induced silencing complex-bound small interfering RNA is a determinant of RNA interference-mediated gene silencing in mice Mol. Pharmacol. 2011, 79 (6) 953-63
6. Whitehead, K. A.; Langer, R.; Anderson, D. G. Knocking down barriers: advances in siRNA delivery Nat. Rev. Drug Discovery 2009, 8 (2) 129-38
7. Dominska, M.; Dykxhoorn, D. M. Breaking down the barriers: siRNA delivery and endosome escape J. Cell Sci. 2010, 123 (Part 8) 1183-9
8. Singh, S.; Narang, A. S.; Mahato, R. I. Subcellular fate and off-target effects of siRNA, shRNA, and miRNA Pharm. Res. 2011, 28 (12) 2996-3015
9. Juliano, R. L.; Ming, X.; Nakagawa, O. Cellular uptake and intracellular trafficking of antisense and siRNA oligonucleotides Bioconjugate Chem. 2012, 23 (2) 147-57
10. Kole, R.; Krainer, A. R.; Altman, S. RNA therapeutics: beyond RNA interference and antisense oligonucleotides Nat. Rev. Drug Discovery 2012, 11 (2) 125-40
11. Zhang, S.; Zhi, D.; Huang, L. Lipid-based vectors for siRNA delivery J. Drug Targeting 2012, 20 (9) 724-35
12. Bartlett, D. W.; Davis, M. E. Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging Nucleic Acids Res. 2006, 34 (1) 322-33
13. 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) 171-82
14. Basha, G. Influence of cationic lipid composition on gene silencing properties of lipid nanoparticle formulations of siRNA in antigen-presenting cells Mol. Ther. 2011, 19 (12) 2186-200
15. Quistorff, B.; Dich, J.; Grunnet, N. Preparation of isolated rat liver hepatocytes Methods Mol. Biol. 1990, 5, 151-60
16. Ryan, N. M. Subcellular fractionation of animal tissues Methods Mol. Biol. 1996, 59, 49-56
17. Pei, Y. Quantitative evaluation of siRNA delivery in vivo RNA 2010, 16 (12) 2553-63
18. Davies, B.; Morris, T. Physiological parameters in laboratory animals and humans Pharm. Res. 1993, 10 (7) 1093-5
19. Ambegia, E. Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression Biochim. Biophys. Acta 2005, 1669 (2) 155-63
20. Semple, S. C. Rational design of cationic lipids for siRNA delivery Nat. Biotechnol. 2010, 28 (2) 172-6
21. Xu, Y.; Koeplinger, K.; Stroh, M.; Aslamkhan, A.; Meacham, D. A.; Carlini, E.; Shi, B.; Matter, A.; Tao, W.; Pei, Y.; Hancock, P.; Marc, A.; Koser, M.; Lyman, M.; Murphy, M.; Stanton, M.; Raab, R.; Hochman, J. Small interfering RNA (siRNA) delivery efficiency evaluation and PK/PD relationship exploration AAPS J. 2011, 13 (Suppl. 2) Abstract R6393
22. Evans, W. H., Ed. Biological Membranes: A Practical Approach; The Practical Approach Series; Findlay, J.B. C.; Evans, W. H., Eds.; Oxford University Press: USA, 1987.
23. Murayama, K. One-step subcellular fractionation of rat liver tissue using a Nycodenz density gradient prepared by freezing-thawing and two-dimensional sodium dodecyl sulfate electrophoresis profiles of the main fraction of organelles Electrophoresis 2001, 22 (14) 2872-80
24. Huotari, J.; Helenius, A. Endosome maturation EMBO J. 2011, 30 (17) 3481-500
25. Sahay, G. Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling Nat. Biotechnol. 2013, 31 (7) 653-8
26. Gilleron, J. Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape Nat. Biotechnol. 2013, 31 (7) 638-46
27. Weitzer, S.; Martinez, J. The human RNA kinase hClp1 is active on 3 transfer RNA exons and short interfering RNAs Nature 2007, 447 (7141) 222-6
28. Meister, G. Human Argonaute2 mediates RNA cleavage targeted by miRNAs and siRNAs Mol. Cell 2004, 15 (2) 185-97
29. Wang, B. Distinct passenger strand and mRNA cleavage activities of human Argonaute proteins Nat. Struct. Mol. Biol. 2009, 16 (12) 1259-66
30. Grimm, D. Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver J. Clin. Invest. 2010, 120 (9) 3106-19