Insight into mechanisms of cellular uptake of lipid nanoparticles and intracellular release of small RNAs

Pharmaceutical Research

Volumn 31, Issue 10, 2014, Pages 2685-2695

  Yu, Bo ^a,b   Wang, Xinmei ^b   Zhou, Chenguang ^b   Teng, Lesheng ^b,c   Ren, Wei ^b   Yang, Zhaogang ^b   Shih, Chih Hsin ^d   Wang, Tianyou ^e   Lee, Robert J ^b,c,g   Tang, Suoqin ^f   Lee, L James ^a,b  

^a Ohio State University   (United States)

^b OHIO STATE UNIVERSITY   (United States)

^c JILIN UNIVERSITY   (China)

^d FENG CHIA UNIVERSITY   (Taiwan)

^e CAPITAL MEDICAL UNIVERSITY   (China)

^f CHINESE PLA GENERAL HOSPITAL   (China)

^g Department of Hematology Capital Institute of Pediatrics Affiliated Children's Hospital ^*  (United States)

Author keywords

intracellular trafficking; lipid nanoparticles; miRNA; molecular beacon; siRNA

Indexed keywords

DIOCTYLDIMETHYL AMMONIUM CHLORIDE; DRUG CARRIER; LIPID; MICRORNA; MIRN122 MICRORNA, HUMAN; N-(1-(2,3-DIOLEYLOXY)PROPYL)-N,N,N-TRIMETHYLAMMONIUM; NANOPARTICLE; QUATERNARY AMMONIUM DERIVATIVE; SMALL INTERFERING RNA;

CHEMISTRY; CONFOCAL MICROSCOPY; DRUG EFFECTS; DRUG FORMULATION; DRUG RELEASE; ENDOCYTOSIS; ENDOSOME; GENETICS; HUMAN; METABOLISM; PARTICLE SIZE; RNA INTERFERENCE; SURFACE PROPERTY; TUMOR CELL LINE;

CELL LINE, TUMOR; DRUG CARRIERS; DRUG COMPOUNDING; DRUG LIBERATION; ENDOCYTOSIS; ENDOSOMES; HUMANS; LIPIDS; MICRORNAS; MICROSCOPY, CONFOCAL; NANOPARTICLES; PARTICLE SIZE; QUATERNARY AMMONIUM COMPOUNDS; RNA INTERFERENCE; RNA, SMALL INTERFERING; SURFACE PROPERTIES;

EID: 84931084545     PISSN: 07248741     EISSN: 1573904X     Source Type: Journal    
DOI: 10.1007/s11095-014-1366-7     Document Type: Article

References (36)

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. 1998;391:806-11.
2. Yu B, Zhao X, Lee LJ, Lee RJ. Targeted delivery systems for oligonucleotide therapeutics. AAPS J. 2009;11:195-203.
3. Pecot CV, Calin GA, Coleman RL, Lopez-Berestein G, Sood AK. RNA interference in the clinic: challenges and future directions. Nat Rev Cancer. 2011;11:59-67.
4. Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference. Nat Rev Genet. 2007;8:173-84.
5. Love KT, Mahon KP, Levins CG, et al. Lipid-like materials for low-dose, in vivo gene silencing. Proc Natl Acad Sci U S A. 2010;107:1864-9.
6. Semple SC, Akinc A, Chen J, et al. Rational design of cationic lipids for siRNA delivery. Nat Biotechnol. 2010;28:172-6.
7. Yu B, Hsu SH, Zhou C, et al. Lipid nanoparticles for hepatic delivery of small interfering RNA. Biomaterials. 2012;33:5924-34.
8. Wang X, Yu B, Ren W, Mo X, Zhou C, He H, et al. Enhanced hepatic delivery of siRNA and microRNA using oleic acid based lipid nanoparticle formulations. J Control Release. 2013;172:690-8.
9. Lin PJ, Tam YY, Hafez I, et al. Influence of cationic lipid composition on uptake and intracellular processing of lipid nanoparticle formulations of siRNA. Nanomedicine. 2013;9:233-46.
10. Wang X, Yu B, Wu Y, Lee RJ, Lee LJ. Efficient down-regulation of CDK4 by novel lipid nanoparticle-mediated siRNA delivery. Anticancer Res. 2011;31:1619-26.
11. Sahay G, Alakhova DY, Kabanov AV. Endocytosis of nanomedicines. J Control Release. 2010;145:182-95.
12. Duncan R, Richardson SC. Endocytosis and intracellular trafficking as gateways for nanomedicine delivery: opportunities and challenges. Mol Pharm. 2012;9:2380-402.
13. Hsu CY, Uludag H. Cellular uptake pathways of lipid-modified cationic polymers in gene delivery to primary cells. Biomaterials. 2012;33:7834-48.
14. Raemdonck K, Remaut K, Lucas B, Sanders NN, Demeester J, De Smedt SC. In situ analysis of single-stranded and duplex siRNA integrity in living cells. Biochemistry. 2006;45:10614-23.
15. Chen HH, Ho YP, Jiang X, Mao HQ, Wang TH, Leong KW. Quantitative comparison of intracellular unpacking kinetics of polyplexes by a model constructed from quantum dot-FRET. Mol Ther. 2008;16:324-32.
16. Abrams MT, Koser ML, Seitzer J, et al. Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: effect of dexamethasone co-treatment. Mol Ther. 2010;18:171-80.
17. Jiang S, Zhang Y. Upconversion nanoparticle-based FRET system for study of siRNA in live cells. Langmuir. 2010;26:6689-94.
18. Shi B, Keough E, Matter A, et al. Biodistribution of small interfering RNA at the organ and cellular levels after lipid nanoparticle-mediated delivery. J Histochem Cytochem. 2011;59:727-40.
19. Alabi CA, Love KT, Sahay G, et al. FRET-labeled siRNA probes for tracking assembly and disassembly of siRNA nanocomplexes. ACS Nano. 2012;6:6133-41.
20. Hirsch M, Strand D, Helm M. Dye selection for live cell imaging of intact siRNA. Biol Chem. 2012;393:23-35.
21. Zhou C, Zhang Y, Yu B, Phelps MA, Lee LJ, Lee RJ. Comparative cellular pharmacokinetics and pharmacodynamics of siRNA delivery by SPANosomes and by cationic liposomes. Nanomedicine. 2013;9:504-13.
22. Jarve A, Muller J, Kim IH, et al. Surveillance of siRNA integrity by FRET imaging. Nucleic Acids Res. 2007;35:e124.
23. Schneider S, Lenz D, Holzer M, Palme K, Suss R. Intracellular FRET analysis of lipid/DNA complexes using flow cytometry and fluorescence imaging techniques. J Control Release. 2010;145:289-96.
24. Shaheen SM, Akita H, Yamashita A, et al. Quantitative analysis of condensation/decondensation status of pDNA in the nuclear sub-domains by QD-FRET. Nucleic Acids Res. 2011;39:e48.
25. Shin S, Kwon HM, Yoon KS, Kim DE, Hah SS. FRET-based probing to gain direct information on siRNA sustainability in live cells: Asymmetric degradation of siRNA strands. Mol Biosyst. 2011;7:2110-3.
26. Hayashi Y, Noguchi Y, Harashima H. Non-linear pharmacokinetics of octaarginine-modified lipid nanoparticles: barriers from in vitro to in vivo. J Control Release. 2012;161:757-62.
27. Yang X, Koh CG, Liu S, et al. Transferrin receptor-targeted lipid nanoparticles for delivery of an antisense oligodeoxyribonucleotide against Bcl-2. Mol Pharm. 2009;6:221-30.
28. Hsu SH, Yu B, Wang X, et al. Cationic lipid nanoparticles for therapeutic delivery of siRNA and miRNA to murine liver tumor. Nanomedicine. 2013;9:1169-80.
29. Wu Y, Ho YP, Mao Y, et al. Uptake and intracellular fate of multifunctional nanoparticles: a comparison between lipoplexes and polyplexes via quantum dot mediated Forster resonance energy transfer. Mol Pharm. 2011;8:1662-8.
30. Wang B, Hsu SH, Wang X, Kutay H, Bid HK, Yu J, et al. Reciprocal regulation of miR-122 and c-Myc in hepatocellular cancer: Role of E2F1 and TFDP2. Hepatology. 2014;59:555-66.
31. Bai S, Nasser MW, Wang B, et al. MicroRNA-122 inhibits tumorigenic properties of hepatocellular carcinoma cells and sensitizes these cells to sorafenib. J Biol Chem. 2009;284:32015-27.
32. Hsu SH, Wang B, Kota J, et al. Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver. J Clin Invest. 2012;122:2871-83.
33. Whitehead KA, Sahay G, Li GZ, et al. Synergistic silencing: combinations of lipid-like materials for efficacious siRNA delivery. Mol Ther. 2011;19:1688-94.
34. Dominska M, Dykxhoorn DM. Breaking down the barriers: siRNA delivery and endosome escape. J Cell Sci. 2010;123:1183-9.
35. Stanton MG, Colletti SL. Medicinal chemistry of siRNA delivery. J Med Chem. 2010;53:7887-901.
36. Garzon R, Marcucci G, Croce CM. Targeting microRNAs in cancer: rationale, strategies and challenges. Nat Rev Drug Discov. 2010;9:775-89.