Bioengineered vaults: Self-assembling protein shell-lipophilic core nanoparticles for drug delivery

ACS Nano

Volumn 8, Issue 8, 2014, Pages 7723-7732

  Buehler, Daniel C ^a,d   Marsden, Matthew D ^b   Shen, Sean ^b   Toso, Daniel B ^b   Wu, Xiaomeng ^b   Loo, Joseph A ^a,b,c   Zhou, Z Hong ^b   Kickhoefer, Valerie A ^a   Wender, Paul A ^d   Zack, Jerome A ^b   Rome, Leonard H ^a,b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d STANFORD UNIVERSITY   (United States)

Author keywords

bryostatin 1; drug delivery systems; HIV latency; nanoparticles; vaults

Indexed keywords

DISEASES; MAMMALS; NANOPARTICLES; RECOMBINANT PROTEINS; TARGETED DRUG DELIVERY; VIRUSES;

ALL-TRANS RETINOIC ACIDS; BRYOSTATIN 1; DRUG DELIVERY SYSTEM; HIV LATENCY; INTRAVENOUS INJECTIONS; NONSTRUCTURAL PROTEINS; THERAPEUTIC COMPOUNDS; VAULTS;

CONTROLLED DRUG DELIVERY;

BRYOSTATIN; BRYOSTATIN 1; DRUG CARRIER; NANOPARTICLE; RIBONUCLEOPROTEIN;

ANIMAL; BIOENGINEERING; CELL LINE; CHEMICAL PHENOMENA; CHEMICAL STRUCTURE; CHEMISTRY; HUMAN; MOUSE; PROTEIN SECONDARY STRUCTURE;

ANIMALS; BIOENGINEERING; BRYOSTATINS; CELL LINE; DRUG CARRIERS; HUMANS; HYDROPHOBIC AND HYDROPHILIC INTERACTIONS; MICE; MODELS, MOLECULAR; NANOPARTICLES; PROTEIN STRUCTURE, SECONDARY; VAULT RIBONUCLEOPROTEIN PARTICLES;

EID: 84906672521     PISSN: 19360851     EISSN: 1936086X     Source Type: Journal    
DOI: 10.1021/nn5002694     Document Type: Article

References (46)

1. Fauman, E. B.; Rai, B. K.; Huang, E. S. Structure-Based Druggability Assessment-Identifying Suitable Targets for Small Molecule Therapeutics Curr. Opin. Chem. Biol. 2011, 15, 463-468
2. Uskokovic, V. Entering the Era of Nanoscience: Time To Be So Small J. Biomed. Nanotechnol. 2013, 9, 1441-1470
3. Ma, Y.; Nolte, R. J.; Cornelissen, J. J. Virus-Based Nanocarriers for Drug Delivery Adv. Drug Delivery Rev. 2012, 64, 811-825
4. Allen, T. M.; Cullis, P. R. Drug Delivery Systems: Entering the Mainstream Science 2004, 303, 1818-1822
5. Felnerova, D.; Viret, J. F.; Gluck, R.; Moser, C. Liposomes and Virosomes as Delivery Systems for Antigens, Nucleic Acids and Drugs Curr. Opin. Biotechnol. 2004, 15, 518-529
6. Duncan, R. The Dawning Era of Polymer Therapeutics Nat. Rev. Drug Discovery 2003, 2, 347-360
7. Rome, L. H.; Kickhoefer, V. A. Development of the Vault Particle as a Platform Technology ACS Nano 2013, 7, 889-902
8. van Zon, A.; Mossink, M. H.; Scheper, R. J.; Sonneveld, P.; Wiemer, E. A. The Vault Complex Cell. Mol. Life Sci. 2003, 60, 1828-1837
9. Suprenant, K. A. Vault Ribonucleoprotein Particles: Sarcophagi, Gondolas, or Safety Deposit Boxes? Biochemistry 2002, 41, 14447-14454
10. Kedersha, N. L.; Rome, L. H. Isolation and Characterization of a Novel Ribonucleoprotein Particle: Large Structures Contain a Single Species of Small RNA J. Cell Biol. 1986, 103, 699-709
11. Tanaka, H.; Kato, K.; Yamashita, E.; Sumizawa, T.; Zhou, Y.; Yao, M.; Iwasaki, K.; Yoshimura, M.; Tsukihara, T. The Structure of Rat Liver Vault at 3.5 Angstrom Resolution Science 2009, 323, 384-388
12. Kedersha, N. L.; Heuser, J. E.; Chugani, D. C.; Rome, L. H. Vaults. III. Vault Ribonucleoprotein Particles Open into Flower-Like Structures with Octagonal Symmetry J. Cell Biol. 1991, 112, 225-235
13. Stephen, A. G.; Raval-Fernandes, S.; Huynh, T.; Torres, M.; Kickhoefer, V. A.; Rome, L. H. Assembly of Vault-Like Particles in Insect Cells Expressing Only the Major Vault Protein J. Biol. Chem. 2001, 276, 23217-23220
14. Kong, L. B.; Siva, A. C.; Rome, L. H.; Stewart, P. L. Structure of the Vault, a Ubiquitous Cellular Component Structure 1999, 7, 371-379
15. Mikyas, Y.; Makabi, M.; Raval-Fernandes, S.; Harrington, L.; Kickhoefer, V. A.; Rome, L. H.; Stewart, P. L. Cryoelectron Microscopy Imaging of Recombinant and Tissue Derived Vaults: Localization of the MVP N-Termini and VPARP J. Mol. Biol. 2004, 344, 91-105
16. Kickhoefer, V. A.; Garcia, Y.; Mikyas, Y.; Johansson, E.; Zhou, J. C.; Raval-Fernandes, S.; Minoofar, P.; Zink, J. I.; Dunn, B.; Stewart, P. L. et al. H. Engineering of Vault Nanocapsules with Enzymatic and Fluorescent Properties Proc. Natl. Acad. Sci. U.S.A. 2005, 102, 4348-4352
17. Poderycki, M. J.; Kickhoefer, V. A.; Kaddis, C. S.; Raval-Fernandes, S.; Johansson, E.; Zink, J. I.; Loo, J. A.; Rome, L. H. The Vault Exterior Shell Is a Dynamic Structure that Allows Incorporation of Vault-Associated Proteins into Its Interior Biochemistry 2006, 45, 12184-12193
18. Bayburt, T. H.; Sligar, S. G. Membrane Protein Assembly into Nanodiscs FEBS Lett. 2010, 584, 1721-1727
19. Civjan, N. R.; Bayburt, T. H.; Schuler, M. A.; Sligar, S. G. Direct Solubilization of Heterologously Expressed Membrane Proteins by Incorporation into Nanoscale Lipid Blayers BioTechniques 2003, 35, 556-560 562-563
20. Ryan, R. O. Nanodisks: Hydrophobic Drug Delivery Vehicles Expert Opin. Drug Delivery 2008, 5, 343-351
21. Tufteland, M.; Ren, G.; Ryan, R. O. Nanodisks Derived from Amphotericin B Lipid Complex J. Pharm. Sci. 2008, 97, 4425-4432
22. Buehler, D. C.; Toso, D. B.; Kickhoefer, V. A.; Zhou, Z. H.; Rome, L. H. Vaults Engineered for Hydrophobic Drug Delivery Small 2011, 7, 1432-1439
23. Erridge, C.; Bennett-Guerrero, E.; Poxton, I. R. Structure and Function of Lipopolysaccharides Microbes Infect. 2002, 4, 837-851
24. Pawlotsky, J. M.; Germanidis, G. The Non-Structural 5A Protein of Hepatitis C Virus J. Viral Hepat. 1999, 6, 343-356
25. Macdonald, A.; Harris, M. Hepatitis C Virus NS5A: Tales of a Promiscuous Protein J. Gen. Virol. 2004, 85, 2485-2502
26. McLauchlan, J. Hepatitis C virus: Viral Proteins on the Move Biochem. Soc. Trans. 2009, 37, 986-990
27. Khabar, K. S.; Polyak, S. J. Hepatitis C Virus-Host Interactions: The NS5A Protein and the Interferon/Chemokine Systems J. Interferon Cytokine Res. 2002, 22, 1005-1012
28. Penin, F.; Brass, V.; Appel, N.; Ramboarina, S.; Montserret, R.; Ficheux, D.; Blum, H. E.; Bartenschlager, R.; Moradpour, D. Structure and Function of the Membrane Anchor Domain of Hepatitis C Virus Nonstructural Protein 5A J. Biol. Chem. 2004, 279, 40835-40843
29. Moradpour, D.; Brass, V.; Penin, F. Function Follows Form: The Structure of the N-Terminal Domain of HCV NS5A Hepatology 2005, 42, 732-735
30. Brass, V.; Bieck, E.; Montserret, R.; Wolk, B.; Hellings, J. A.; Blum, H. E.; Penin, F.; Moradpour, D. An Amino-Terminal Amphipathic Alpha-Helix Mediates Membrane Association of the Hepatitis C Virus Nonstructural Protein 5A J. Biol. Chem. 2002, 277, 8130-8139
31. Tellinghuisen, T. L.; Marcotrigiano, J.; Rice, C. M. Structure of the Zinc-Binding Domain of an Essential Component of the Hepatitis C Virus Replicase Nature 2005, 435, 374-379
32. Hu, H.; Larson, R. G. Preparation of Fluorescent Particles with Long Excitation and Emission Wavelengths Dispersible in Organic Solvents Langmuir 2004, 20, 7436-7443
33. Zeman, S. M.; Phillips, D. R.; Crothers, D. M. Characterization of Covalent Adriamycin-DNA Adducts Proc. Natl. Acad. Sci. U.S.A. 1998, 95, 11561-11565
34. Hamill, R. J. Amphotericin B Formulations: A Comparative Review of Efficacy and Toxicity Drugs 2013, 73, 919-934
35. Mehla, R.; Bivalkar-Mehla, S.; Zhang, R.; Handy, I.; Albrecht, H.; Giri, S.; Nagarkatti, P.; Nagarkatti, M.; Chauhan, A. Bryostatin Modulates Latent HIV-1 Infection via PKC and AMPK Signaling but Inhibits Acute Infection in a Receptor Independent Manner PLoS One 2010, 5, e11160
36. DeChristopher, B. A.; Loy, B. A.; Marsden, M. D.; Schrier, A. J.; Zack, J. A.; Wender, P. A. Designed, Synthetically Accessible Bryostatin Analogues Potently Induce Activation of Latent HIV Reservoirs in Vitro Nat. Chem. 2012, 4, 705-710
37. Wender, P. A.; Baryza, J. L.; Brenner, S. E.; DeChristopher, B. A.; Loy, B. A.; Schrier, A. J.; Verma, V. A. Design, Synthesis, and Evaluation of Potent Bryostatin Analogs that Modulate PKC Translocation Selectivity Proc. Natl. Acad. Sci. U.S.A. 2011, 108, 6721-6726
38. Marsden, M. D.; Zack, J. A. Establishment and Maintenance of HIV Latency: Model Systems and Opportunities for Intervention Future Virol. 2010, 5, 97-109
39. Marsden, M. D.; Zack, J. A. HIV/AIDS Eradication Bioorg. Med. Chem. Lett. 2013, 23, 4003-4010
40. Kovochich, M.; Marsden, M. D.; Zack, J. A. Activation of Latent HIV Using Drug-Loaded Nanoparticles PLoS One 2011, 6, e18270
41. Bignami, G. S.; Wagner, F.; Grothaus, P. G.; Rustagi, P.; Davis, D. E.; Kraft, A. S. Biological Activity of 26-Succinylbryostatin 1 Biochim. Biophys. Acta 1996, 1312, 197-206
42. Nelson, T. J.; Sen, A.; Alkon, D. L.; Sun, M. K. Adduct Formation in Liquid Chromatography-Triple Quadrupole Mass Spectrometric Measurement of Bryostatin 1 J. Chromatogr. B Analyt. Technol. Biomed. Life Sci. 2013, 944C, 55-62
43. Beans, E. J.; Fournogerakis, D.; Gauntlett, C.; Heumann, L. V.; Kramer, R.; Marsden, M. D.; Murray, D.; Chun, T. W.; Zack, J. A.; Wender, P. A. Highly Potent, Synthetically Accessible Prostratin Analogs Induce Latent HIV Expression in Vitro and ex Vivo Proc. Natl. Acad. Sci. U.S.A. 2013, 110, 11698-11703
44. Bear, H. D.; McFadden, A. W.; Kostuchenko, P. J.; Lipshy, K. A.; Hamad, G. G.; Turner, A. J.; Roberts, J. D.; Carr, M.; Carr, S.; Grant, S. Bryostatin 1 Activates Splenic Lymphocytes and Induces Sustained Depletion of Splenocyte Protein Kinase C Activity in Vivo after a Single Intravenous Administration Anticancer Drugs 1996, 7, 299-306
45. Kickhoefer, V. A.; Han, M.; Raval-Fernandes, S.; Poderycki, M. J.; Moniz, R. J.; Vaccari, D.; Silvestry, M.; Stewart, P. L.; Kelly, K. A.; Rome, L. H. Targeting Vault Nanoparticles to Specific Cell Surface Receptors ACS Nano 2009, 3, 27-36
46. Wender, P. A.; Kee, J. M.; Warrington, J. M. Practical Synthesis of Prostratin, DPP, and Their Analogs, Adjuvant Leads against Latent HIV Science 2008, 320, 649-652