In vitro - In vivo translation of lipid nanoparticles for hepatocellular siRNA delivery

ACS Nano

Volumn 6, Issue 8, 2012, Pages 6922-6929

  Whitehead, Kathryn A ^a   Matthews, Jonathan ^a   Chang, Philip H ^a   Niroui, Farnaz ^a   Dorkin, J Robert ^a   Severgnini, Mariano ^b   Anderson, Daniel G ^a,c  

^a MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

^b ALNYLAM PHARMACEUTICALS   (United States)

^c Massachusetts Institute of Technology Cambridge   (United States)

Author keywords

lipidoids; liposomes; liver delivery; nanoparticles; siRNA delivery

Indexed keywords

DATA SETS; DELIVERY VEHICLE; ENTRAPMENT EFFICIENCY; EXPERIMENTAL SYSTEM; GUIDING PRINCIPLES; HELA CELL; HEPATOCELLULAR; HIGH-THROUGHPUT TECHNIQUE; IN-VITRO; IN-VIVO; LIPID NANOPARTICLES; LIPIDOIDS; NANOPARTICLE SIZES; PRIMARY HEPATOCYTES; SIRNA DELIVERY; TRANSFECTION EFFICIENCY; TRANSLATABILITY;

ANIMAL CELL CULTURE; ANIMALS; LIPOSOMES; RNA;

NANOPARTICLES;

LIPID; NANOCAPSULE; SMALL INTERFERING RNA;

ANIMAL; ARTICLE; CHEMISTRY; GENE SILENCING; GENETIC TRANSFECTION; GENETICS; HELA CELL; HUMAN; LIVER CELL CARCINOMA; METHODOLOGY; MOUSE; PARTICLE SIZE; SURFACE PROPERTY; TUMOR CELL LINE; ULTRASTRUCTURE;

ANIMALS; CARCINOMA, HEPATOCELLULAR; CELL LINE, TUMOR; GENE SILENCING; HELA CELLS; HUMANS; LIPIDS; MICE; NANOCAPSULES; PARTICLE SIZE; RNA, SMALL INTERFERING; SURFACE PROPERTIES; TRANSFECTION;

EID: 84865584957     PISSN: 19360851     EISSN: 1936086X     Source Type: Journal    
DOI: 10.1021/nn301922x     Document Type: Article

References (39)

1. Metz, S.; Haas, A. K.; Daub, K.; Croasdale, R.; Stracke, J.; Lau, W.; Georges, G.; Josel, H.-P.; Dziadek, S.; Hopfner, K.-P. et al. Bispecific Digoxigenin-Binding Antibodies for Targeted Payload Delivery Proc. Natl. Acad. Sci. U.S.A. 2011, 108, 8194-8199
2. Lehto, T.; Simonson, O. E.; Mäger, I.; Ezzat, K.; Sork, H.; Copolovici, D.-M.; Viola, J. R.; Zaghloul, E. M.; Lundin, P.; Moreno, P. M. D. et al. A Peptide-Based Vector for Efficient Gene Transfer in Vitro and in Vivo Mol. Ther. 2011, 19, 1457-1467
3. Marrero, B.; Heller, R. The Use of an In Vitro 3D Melanoma Model To Predict In Vivo Plasmid Transfection Using Electroporation Biomaterials 2012, 33, 3036-3046
4. Correia, C.; Grayson, W. L.; Park, M.; Hutton, D.; Zhou, B.; Guo, X. E.; Niklason, L.; Sousa, R. A.; Reis, R. L.; Vunjak-Novakovic, G. In Vitro Model of Vascularized Bone: Synergizing Vascular Development and Osteogenesis PLoS ONE 2011, 6, e28352
5. Yamada, K. M.; Cukierman, E. Modeling Tissue Morphogenesis and Cancer in 3D Cell 2007, 130, 601-610
6. Khetani, S. R.; Bhatia, S. N. Microscale Culture of Human Liver Cells for Drug Development Nat. Biotechnol. 2007, 26, 120-126
7. Pecot, C. V.; Calin, G. A.; Coleman, R. L.; Lopez-Berestein, G.; Sood, A. K. RNA Interference in the Clinic: Challenges and Future Directions Nat. Rev. Cancer 2010, 11, 59-67
8. Shim, M. S.; Kwon, Y. J. Efficient and Targeted Delivery of siRNA in Vivo FEBS J. 2010, 277, 4814-4827
9. Akinc, A.; Zumbuehl, A.; Goldberg, M.; Leshchiner, E. S.; Busini, V.; Hossain, N.; Bacallado, S. A.; Nguyen, D. N.; Fuller, J.; Alvarez, R. et al. A Combinatorial Library of Lipid-like Materials for Delivery of RNAi Therapeutics Nat. Biotechnol. 2008, 26, 561-569
10. Love, K. T.; Mahon, K. P.; Levins, C. G.; Whitehead, K. A.; Querbes, W.; Dorkin, J. R.; Qin, J.; Cantley, W.; Qin, L. L.; Racie, T. et al. Lipid-like Materials for Low-Dose, In Vivo Gene Silencing Proc. Natl. Acad. Sci. U.S.A. 2010, 107, 1864-1869
11. Mahon, K. P.; Love, K. T.; Whitehead, K. A.; Qin, J.; Akinc, A.; Leshchiner, E.; Leshchiner, I.; Langer, R.; Anderson, D. G. Combinatorial Approach To Determine Functional Group Effects on Lipidoid-Mediated siRNA Delivery Bioconjugate Chem. 2010, 21, 1448-1454
12. Whitehead, K. A.; Sahay, G.; Li, G. Z.; Love, K. T.; Alabi, C. A.; Ma, M.; Zurenko, C.; Querbes, W.; Langer, R. S.; Anderson, D. G. Synergistic Silencing: Combinations of Lipid-like Materials for Efficacious siRNA Delivery Mol. Ther. 2011, 19, 1688-1694
13. Li, S.-D.; Huang, L. Stealth Nanoparticles: High Density but Sheddable PEG Is a Key for Tumor Targeting J. Controlled Release 2010, 145, 178-181
14. Akinc, A.; Querbes, W.; De, S.; Qin, J.; Frank-Kamenetsky, M.; Jayaprakash, K. N.; Jayaraman, M.; Rajeev, K. G.; Cantley, W. L.; Dorkin, J. R. et al. Targeted Delivery of RNAi Therapeutics with Endogenous and Exogenous Ligand-Based Mechanisms Mol. Ther. 2009, 18, 1357-1364
15. 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. et al. Evaluation of Efficacy, Biodistribution, and Inflammation for a Potent siRNA Nanoparticle: Effect of Dexamethasone Co-treatment Mol. Ther. 2009, 1-10
16. 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, 1067-1070
17. Schroeder, A.; Dahlman, J. E.; Sahay, G.; Love, K. T.; Jiang, S.; Eltoukhy, A. A.; Levins, C. G.; Wang, Y.; Anderson, D. G. Alkane-Modified Short Polyethyleneimine for siRNA Delivery. J. Controlled Release 2011, not supplied.
18. Varkouhi, A. K.; Mountrichas, G.; Schiffelers, R. M.; Lammers, T.; Storm, G.; Pispas, S.; Hennink, W. E. Polyplexes Based on Cationic Polymers with Strong Nucleic Acid Binding Properties Eur. J. Pharm. Sci. 2012, 45, 459-466
19. Masson, C.; Garinot, M.; Mignet, N.; Wetzer, B.; Mailhe, P.; Scherman, D.; Bessodes, M. pH-Sensitive PEG Lipids Containing Orthoester Linkers: New Potential Tools for Nonviral Gene Delivery J. Controlled Release 2004, 99, 423-434
20. Barichello, J. M.; Kizuki, S.; Tagami, T.; Asai, T.; Ishida, T.; Kikuchi, H.; Oku, N.; Kiwada, H. Agitation during Lipoplex Formation Improves the Gene Knockdown Effect of siRNA Int. J. Pharm. 2011, 410, 153-160
21. Elaut, G.; Henkens, T.; Papeleu, P.; Snykers, S.; Vinken, M.; Vanhaecke, T.; Rogiers, V. Molecular Mechanisms Underlying the Dedifferentiation Process of Isolated Hepatocytes and Their Cultures Curr. Drug Metab. 2006, 7, 629-660
22. Vinken, M.; Decrock, E.; Doktorova, T.; Ramboer, E.; Vuyst, E.; Vanhaecke, T.; Leybaert, L.; Rogiers, V. Characterization of Spontaneous Cell Death in Monolayer Cultures of Primary Hepatocytes Arch. Toxicol. 2011, 85, 1589-1596
23. Rao, S.; Song, Y.; Peddie, F.; Evans, A. M. Particle Size Reduction to the Nanometer Range: A Promising Approach To Improve Buccal Absorption of Poorly Water-Soluble Drugs Int. J. Nanomed. 2011, 6, 1245-1251
24. Kong, G.; Braun, R. D.; Dewhirst, M. W. Hyperthermia Enables Tumor-Specific Nanoparticle Delivery: Effect of Particle Size Cancer Res. 2000, 60, 4440-4445
25. Zhang, M.; Ishii, A.; Nishiyama, N. PEGylated Calcium Phosphate Nanocomposites as Smart Environment-Sensitive Carriers for siRNA Delivery Adv. Mater. 2009, 21, 3520-3525
26. Di Paolo, D.; Brignole, C.; Pastorino, F.; Carosio, R.; Zorzoli, A.; Rossi, M.; Loi, M.; Pagnan, G.; Emionite, L.; Cilli, M. et al. Neuroblastoma-Targeted Nanoparticles Entrapping siRNA Specifically Knockdown ALK Mol. Ther. 2011, 19, 1131-1140
27. Gjetting, T.; Arildsen, N. S.; Christensen, C. L.; Poulsen, T. T.; Roth, J. A.; Handlos, V. N.; Poulsen, H. S. In Vitro and In Vivo Effects of Polyethylene Glycol (PEG)-Modified Lipid in DOTAP/Cholesterol-Mediated Gene Transfection Int. J. Nanomed. 2010, 5, 371-383
28. Lee, S. H.; Mok, H.; Park, T. G. Di- and Triblock siRNA-PEG Copolymers: PEG Density Effect of Polyelectrolyte Complexes on Cellular Uptake and Gene Silencing Efficiency Macromol. Biosci. 2010, 11, 410-418
29. Guillouzo, A. Liver Cell Models in In Vitro Toxicology Environ. Health Perspect. 1998, 106, 511
30. Almeida, J. P. M.; Chen, A. L.; Foster, A.; Drezek, R. In Vivo Biodistribution of Nanoparticles Nanomedicine 2011, 6, 815-835
31. Kundu, A. K.; Chandra, P. K.; Hazari, S.; Pramar, Y. V.; Dash, S.; Mandal, T. K. Development and Optimization of Nanosomal Formulations for siRNA Delivery to the Liver Eur. J. Pharm. Biopharm. 2012, 80, 257-267
32. Johnston, H. J.; Semmler-Behnke, M.; Brown, D. M.; Kreyling, W.; Tran, L.; Stone, V. Evaluating the Uptake and Intracellular Fate of Polystyrene Nanoparticles by Primary and Hepatocyte Cell Lines in Vitro Toxicol. Appl. Pharmacol. 2010, 242, 66-78
33. Wisse, E.; de Zanger, R. B.; Charels, K.; van der Smissen, P.; McCuskey, R. S. The Liver Sieve: Considerations Concerning the Structure and Function of Endothelial Fenestrae, the Sinusoidal Wall and the Space of Disse Hepatology 1985, 5, 683-692
34. Kim, S. I.; Shin, D.; Choi, T. H.; Lee, J. C.; Cheon, G.-J.; Kim, K.-Y.; Park, M.; Kim, M. Systemic and Specific Delivery of Small Interfering RNAs to the Liver Mediated by Apolipoprotein a-I Molecular Therapy 2007, 15, 1145-1152
35. Lenaerts, V.; Nagelkerke, J. F.; Van Berkel, T. J. C.; Couvreur, P.; Grislain, L.; Roland, M.; Speiser, P. In Vivo Uptake of Polyisobutyl Cyanoacrylate Nanoparticles by Rat Liver Kupffer, Endothelial, and Parenchymal Cells J. Pharm. Sci. 1984, 73, 980-982
36. Hayashi, Y.; Yamauchi, J.; Khalil, I. A.; Kajimoto, K.; Akita, H.; Harashima, H. Cell Penetrating Peptide-Mediated Systemic siRNA Delivery to the Liver Int. J. Pharm. 2011, 419, 308-313
37. Zuckerman, J. E.; Choi, C. H. J.; Han, H.; Davis, M. E. Polycation-siRNA Nanoparticles Can Disassemble at the Kidney Glomerular Basement Membrane Proc. Natl. Acad. Sci. U.S.A. 2012, 109, 3137-3142
38. Yang, S.; Delgado, R.; Barker, C. S.; Yang, Z.-Y.; Nabel, G. J.; King, S. R.; Woffendin, C.; Xu, L.; Nolan, G. P. Generation of Retroviral Vector for Clinical Studies Using Transient Transfection Hum. Gene Ther. 1999, 10, 123-132
39. Severgnini, M.; Sherman, J.; Sehgal, A.; Jayaprakash, N. K.; Aubin, J.; Wang, G.; Zhang, L.; Peng, C. G.; Yucius, K.; Butler, J. et al. A Rapid Two-Step Method for Isolation of Functional Primary Mouse Hepatocytes: Cell Characterization and Asialoglycoprotein Receptor Based Assay Development Cytotechnology 2011, 64, 187-195