Liposomes as siRNA delivery vectors

Current Drug Metabolism

Volumn 15, Issue 9, 2014, Pages 882-892

  Bochicchio, Sabrina ^a   Dalmoro, Annalisa ^a   Barba, Anna Angela ^a   Grassi, Gabriele ^b,c   Lamberti, Gaetano ^a  

^a UNIVERSITY OF SALERNO   (Italy)

^b UNIVERSITY OF TRIESTE   (Italy)

^c UNIVERSITY OF TRIESTE   (Italy)

Author keywords

Drug delivery; Encapsulating methods; Liposomes; Nucleic acid based drugs (NABDs); Polymeric carriers; Small interfering RNAs (siRNAs)

Indexed keywords

ALCOHOL; DETERGENT; LIPID; LIPOSOME; ORGANIC SOLVENT; SMALL INTERFERING RNA;

DILUTION; DRUG DELIVERY SYSTEM; GENE VECTOR; GENETIC TRANSFECTION; HYDRATION; IN VITRO STUDY; LIPID BILAYER; MEMBRANE PERMEABILITY; NANOENCAPSULATION; NEOPLASM; NONVIRAL GENE DELIVERY SYSTEM; PARTICLE SIZE; REVIEW; RNA INTERFERENCE; ANIMAL; GENE TRANSFER; HUMAN;

ANIMALS; GENE TRANSFER TECHNIQUES; HUMANS; LIPOSOMES; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 84930851710     PISSN: 13892002     EISSN: 18755453     Source Type: Journal    
DOI: 10.2174/1389200216666150206124913     Document Type: Review

References (82)

1. Fire A., Xu S., Montgomery M.K., Kostas S.A., Driver S.E., Mello C.C. Potent and specific genetic interference by doublestranded RNA in Caenorhabditis elegans. Nature 1998 391 806-811.
2. Sledz C.A., Williams B.R. RNA interference in biology and disease. Blood 2005 106 787-794.
3. 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 411 494-498.
4. Lee Y., Ahn C., Han J., Choi H., Kim J., Yim J., Lee J., Provost P., Rådmark O., Kim S. The nuclear RNase III Drosha initiates microRNA processing. Nature 2003 425 415-419.
5. Bernstein E., Caudy A.A., Hammond S.M., Hannon G.J. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature 2001 409 363-366.
6. Novina C.D., Sharp P.A. The rnai revolution. Nature 2004 430 161-164.
7. He L., Hannon G.J. MicroRNAs: small RNAs with a big role in gene regulation. Nat. Rev. Genet., 2004 5 522-531.
8. Wostenberg C., Lary J.W., Sahu D., Acevedo R., Quarles K.A., Cole J.L., Showalter S.A. The Role of Human Dicer-dsRBD in Processing Small Regulatory RNAs. PloS One 2012 7 e51829.
9. Dapas B., Farra R., Grassi M., Giansante C., Fiotti N., Uxa L., Rainaldi G., Mercatanti A., Colombatti A., Spessotto P. Role of E2F1-Cyclin E1-Cyclin E2 Circuit in Human Coronary Smooth Muscle Cell Proliferation and Therapeutic Potential of Its Downregulation by siRNAs. Mol. Med., 2009 15 297.
10. DeVincenzo J., Lambkin-Williams R., Wilkinson T., Cehelsky J., Nochur S., Walsh E., Meyers R., Gollob J., Vaishnaw A. A randomized double-blind placebo-controlled study of an RNAibased therapy directed against respiratory syncytial virus. Proc. Nat. Acad. Sci., 2010 107 8800-8805.
11. Kaiser P.K., Symons R., Shah S.M., Quinlan E.J., Tabandeh H., Do D.V., Reisen G., Lockridge J.A., Short B, Guerciolini R. RNAi-based treatment for neovascular age-related macular degeneration by Sirna-027. Am. J. Ophthalmol., 2010 150 33-39. e2.
12. Watts J.K., Corey D.R. Clinical status of duplex RNA. Bioorg. Med. Chem. Lett., 2010 20 3203-3207.
13. Miller V.M., Gouvion C.M., Davidson B.L., Paulson H.L. Targeting Alzheimer's disease genes with RNA interference: An efficient strategy for silencing mutant alleles. Nucleic Acids Res., 2004 32 661-668.
14. Wang H., Ghosh A., Baigude H., Yang C.-s., Qiu L., Xia X., Zhou H., Rana T.M., Xu Z. Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression. J. Biol. Chem., 2008 283 15845-15852.
15. Wilson J.A., Jayasena S., Khvorova A., Sabatinos S., Rodrigue-Gervais I.G., Arya S., Sarangi F., Harris-Brandts M., Beaulieu S., Richardson C.D. RNA interference blocks gene expression and RNA synthesis from hepatitis C replicons propagated in human liver cells. Proc. Natl. Acad. Sci., 2003 100 2783-2788.
16. Song E., Lee S.-K., Wang J., Ince N., Ouyang N., Min J., Chen J., Shankar P., Lieberman J. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat. Med., 2003 9 347-351.
17. Tabernero J., Shapiro G.I., LoRusso P.M., Cervantes A., Schwartz G.K., Weiss G.J., Paz-Ares L., Cho D.C., Infante J.R., Alsina M. First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement. Cancer Discov., 2013 3 406-417.
18. Scaggiante B., Kazemi M., Pozzato G., Dapas B., Farra R., Grassi M., Zanconati F., Grassi G. Novel hepatocellular carcinoma molecules with prognostic and therapeutic potentials. World J. Gastroenterol., 2014 20 1268-1288.
19. Baiz D., Dapas B., Farra R., Scaggiante B., Pozzato G., Zanconati F., Fiotti N., Consoloni L., Chiaretti S., Grassi G. Bortezomib effect on E2F and cyclin family members in human hepatocellular carcinoma cell lines. World J. Gastroenterol., 2014 20 795-803.
20. Farra R., Dapas B., Pozzato G., Scaggiante B., Agostini F., Zennaro C., Grassi M., Rosso N., Giansante C., Fiotti N. Effects of E2F1-cyclin E1-E2 circuit down regulation in hepatocellular carcinoma cells. Dig. Liver Dis., 2011 43 1006-1014.
21. Novina C.D., Murray M.F., Dykxhoorn D.M., Beresford P.J., Riess J., Lee S.-K., Collman R.G., Lieberman J., Shankar P., Sharp P.A. siRNA-directed inhibition of HIV-1 infection. Nat. Med., 2002 8 681-686.
22. Grassi G., Scaggiante B., Dapas B., Farra R., Tonon F., Lamberti G., Barba A., Fiorentino S., Fiotti N., Zanconati F. Therapeutic Potential of Nucleic Acid-Based Drugs in Coronary Hyper-Proliferative Vascular Diseases. Curr. Med. Chem., 2013.
23. Zanetti M., Stocca A., Dapas B., Farra R., Uxa L., Bosutti A., Barazzoni R., Bossi F., Giansante C., Tedesco F. Inhibitory effects of fenofibrate on apoptosis and cell proliferation in human endothelial cells in high glucose. J. Mol. Med., 2008 86 185-195.
24. David S., Resnier P., Guillot A., Pitard B., Benoit J.-P., Passirani C. siRNA LNCs-A novel platform of lipid nanocapsules for systemic siRNA administration. Eur. J. Pharm. Biopharm., 2012 81 448-452.
25. Miele E., Spinelli G.P., Miele E., Di Fabrizio E., Ferretti E., Tomao S., Gulino A. Nanoparticle-based delivery of small interfering RNA: challenges for cancer therapy. Int. J. Nanomedicine 2012 7 3637.
26. Missailidis S. Future Trends in Cancer. Anticancer Ther., 2008 387.
27. Fattal E., Bochot A. State of the art and perspectives for the delivery of antisense oligonucleotides and siRNA by polymeric nanocarriers. Int. J. Pharmaceut., 2008 364 237-248.
28. Parker A.L., Newman C., Briggs S., Seymour L., Sheridan P.J. Nonviral gene delivery: Techniques and implications for molecular medicine. Exp. Rev. Mol. Med., 2003 2 1-15.
29. Gavrilov K., Saltzman W.M. Therapeutic siRNA: principles challenges and strategies. Yale J. Biol. Med., 2012 85 187.
30. Lee J.H., Lee K., Moon S.H., Lee Y., Park T.G., Cheon J. Allin-One Target-Cell-Specific Magnetic Nanoparticles for Simultaneous Molecular Imaging and siRNA Delivery. Angew. Chem., 2009 121 4238-4243.
31. Kreuter J. Nanoparticles and microparticles for drug and vaccine delivery. J. Anat., 1996 189 503.
32. Freiberg S., Zhu X. Polymer microspheres for controlled drug release. Int. J. Pharmaceut., 2004 282 1-18.
33. Dalmoro A., Lamberti G., Titomanlio G., Barba A.A., d'Amore M. Enteric Micro-Particles for Targeted Oral Drug Delivery. AAPS PharmSciTech., 2010 11 1500-1507.
34. Barba A.A., Dalmoro A., d'Amore M., Lamberti G. Controlled release of drugs from microparticles produced by ultrasonic assisted atomization based on biocompatible polymers. Chem. Biochem. Eng. Q., 2012 26 345-353.
35. Mahapatro A., Singh D.K. Biodegradable nanoparticles are excellent vehicle for site directed in-vivo delivery of drugs and vaccines. J. Nanobiotechnol., 2011 9 55.
36. Vrignaud S., Benoit J.-P., Saulnier P. Strategies for the nanoencapsulation of hydrophilic molecules in polymer-based nanoparticles. Biomaterials 2011 32 8593-8604.
37. Sawant R.R., Torchilin V.P. Liposomes as 'smart'pharmaceutical nanocarriers. Soft Matter 2010 6 4026-4044.
38. Schiffelers R.M., Storm G. Liposomal nanomedicines as anticancer therapeutics: beyond targeting tumor cells. Int. J. Pharmaceut., 2008 364 258-264.
39. Coelho J.F., Ferreira P.C., Alves P., Cordeiro R., Fonseca A.C., Góis J.R., Gil M.H. Drug delivery systems: Advanced technologies potentially applicable in personalized treatments. The EPMA J., 2010 1 164-209.
40. Samad A., Sultana Y., Aqil M. Liposomal drug delivery systems: An update review. Curr. Drug Deliv., 2007 4 297-305.
41. Nagata T., Okada K., Takebe I., Matsui C. Delivery of tobacco mosaic virus RNA into plant protoplasts mediated by reverse-phase evaporation vesicles (liposomes). Mol. Gen. Genet., 1981 184 161-165.
42. Szoka F., Papahadjopoulos D. Procedure for preparation of liposomes with large internal aqueous space and high capture by reverse-phase evaporation. Proc. Natl. Acad. Sci., 1978 75 4194-4198.
43. Sipai Altaf Bhai M., Vandana Y., Prasanth V.V. Liposomes: An overview. JPSI 2012 1 13-21.
44. 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.
45. Yu Y.H., Kim E., Park D.E., Shim G., Lee S., Kim Y.B., Kim C.-W., Oh Y.-K. Cationic solid lipid nanoparticles for co-delivery of paclitaxel and siRNA. Eur. J. Pharm. Biopharm., 2012 80 268-273.
46. Jiang J., Yang S.-j., Wang J.-c., Yang L.-j., Xu Z.-z., Yang T., Liu X.-Y., Zhang Q. Sequential treatment of drug-resistant tumors with RGD-modified liposomes containing siRNA or doxorubicin. Eur. J. Pharm. Biopharm., 2010 76 170-178.
47. Gomes-da-Silva L.C., Ramalho J.S., Pedroso de Lima M.C., Simões S., Moreira J.N. Impact of anti-PLK1 siRNA-containing F3-targeted liposomes on the viability of both cancer and endothelial cells. Eur. J. Pharm. Biopharm., 2013.
48. MacLachlan I. in: Antisense Drug Technology: Principles Strategies and Applications S.T. Crooke Ed., Taylor & Francis 2008, pp. 237-270.
49. Patil S.D., Rhodes D.G., Burgess D.J. DNA-based therapeutics and DNA delivery systems: A comprehensive review. AAPS J., 2005 7 E61-E77.
50. Li Y., Wang J., Gao Y., Zhu J., Wientjes M.G., Au J.L.-S. Relationships between liposome properties cell membrane binding intracellular processing and intracellular bioavailability. AAPS J., 2011 13 585-597.
51. Carmona S., Jorgensen M.R., Kolli S., Crowther C., Salazar F.H., Marion P.L., Fujino M., Natori Y., Thanou M., Arbuthnot P. Controlling HBV replication in vivo by intravenous administration of triggered PEGylated siRNA-nanoparticles. Mol. Pharmaceut., 2009 6 706-717.
52. Ceh B., Winterhalter M., Frederik P.M., Vallner J.J., Lasic D.D. Stealth® liposomes: from theory to product. Adv. Drug Deliv. Rev., 1997 24 165-177.
53. Desigaux L., Sainlos M., Lambert O., Chevre R., Letrou-Bonneval E., Vigneron J.-P., Lehn P., Lehn J.-M., and Pitard B. Self-assembled lamellar complexes of siRNA with lipidic aminoglycoside derivatives promote efficient siRNA delivery and interference. Proc. Nat. Acad. Sci., 2007 104 16534-16539.
54. Buyens K., De Smedt S.C., Braeckmans K., Demeester J., Peeters L., van Grunsven L.A., de Mollerat du Jeu X., Sawant R., Torchilin V., Farkasova K. Liposome based systems for systemic siRNA delivery: stability in blood sets the requirements for optimal carrier design. J. Control. Release 2012 158 362-370.
55. Torchilin V.P. Recent advances with liposomes as pharmaceutical carriers. Nat. Rev. Drug Discov. 2005 4 145-160.
56. Malmsten M. Soft drug delivery systems. Soft Matter 2006 2 760-769.
57. Immordino M.L., Dosio F., Cattel L. Stealth liposomes: review of the basic science rationale and clinical applications existing and potential. Int. J. Nanomedicine 2006 1 297.
58. Podesta J.E., Kostarelos K. Engineering cationic liposome: siRNA complexes for in vitro and in vivo delivery. Methods Enzymol., 2009 464 343-354.
59. Kim S.I., Shin D., Choi T.H., Lee J.C., Cheon G.-J., Kim K.-Y., Park M., and Kim M. Systemic and specific delivery of small interfering RNAs to the liver mediated by apolipoprotein AI. Mol. Ther., 2007 15 1145-1152.
60. Lee H., Kim S.I., Shin D., Yoon Y., Choi T.H., Cheon G.-J., Kim M. Hepatic siRNA delivery using recombinant human apolipoprotein AI in mice. Biochem. Biophys. Res. Commun., 2009 378 192-196.
61. Whitehead K.A., Langer R., Anderson D.G. Knocking down barriers: Advances in siRNA delivery. Nat. Rev. Drug Discov 2009 8 129-138.
62. Kostarelos K., Miller A.D. Synthetic self-assembly ABCD nanoparticles, a structural paradigm for viable synthetic non-viral vectors. Chem. Soc. Rev., 2005 34 970-994.
63. Li S.-D., Huang L. Nanoparticles evading the reticuloendothelial system: role of the supported bilayer. Biochim. Biophys. Acta 2009 1788 2259-2266.
64. Klibanov A.L., Maruyama K., Torchilin V.P., Huang L. Amphipathic polyethyleneglycols effectively prolong the circulation time of liposomes. FEBS Lett., 1990 268 235-237.
65. Kim S.-S., Peer D., Kumar P., Subramanya S., Wu H., Asthana D., Habiro K., Yang Y.-G., Manjunath N., Shimaoka M. RNAimediated CCR5 silencing by LFA-1-targeted nanoparticles prevents HIV infection in BLT mice. Mol. Ther., 2010 18 370-376.
66. Li J., Chen Y.-C., Tseng Y.-C., Mozumdar S., Huang L. Biodegradable calcium phosphate nanoparticle with lipid coating for systemic siRNA delivery. J. Control. Release 2010 142 416-421.
67. Buyens K., Demeester J., De Smedt S.S., Sanders N.N. Elucidating the encapsulation of short interfering RNA in PEGylated cationic liposomes. Langmuir 2009 25 4886-4891.
68. Semple S.C., Klimuk S.K., Harasym T.O., Dos Santos N., Ansell S.M., Wong K.F., Maurer N., Stark H., Cullis P.R., Hope M.J. Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures. Biochim. Biophys. Acta 2001 1510 152-166.
69. Morrissey D.V., Lockridge J.A., Shaw L., Blanchard K., Jensen K., Breen W., Hartsough K., Machemer L., Radka S., Jadhav V. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat. Biotechnol., 2005 23 1002-1007.
70. Akinc A., Goldberg M., Qin J., Dorkin J.R., Gamba-Vitalo C., Maier M., Jayaprakash K.N., Jayaraman M., Rajeev K.G., Manoharan M. Development of lipidoid-siRNA formulations for systemic delivery to the liver. Mol. Ther., 2009 17 872-879.
71. Wu S.Y., McMillan N.A. Lipidic systems for in vivo siRNA delivery. AAPS J., 2009 11 639-652.
72. Barba A.A., Bochicchio S., Lamberti G., Dalmoro A. Ultrasonic energy in liposome production: process modelling and size calculation. Soft Matter 2014 10 2574-2581.
73. Geisbert T.W., Hensley L.E., Kagan E., Yu E.Z., Geisbert J.B., Daddario-DiCaprio K., Fritz E.A., Jahrling P.B., McClintock K., Phelps J.R. Postexposure protection of guinea pigs against a lethal ebola virus challenge is conferred by RNA interference. J. Infect. Dis., 2006 193 1650-1657.
74. Geisbert T.W., Lee A.C., Robbins M., Geisbert J.B., Honko A.N., Sood V., Johnson J.C., de Jong S., Tavakoli I., Judge A. Postexposure protection of non-human primates against a lethal Ebola virus challenge with RNA interference: A proof-of-concept study. The Lancet 2010 375 1896-1905.
75. MacLachlan I. Progress in the Development of Lipid Nanoparticle-RNA Therapeutics. in ASGCT 17th Annual Meeting. 2014. Washington DC.
76. Santel A., Aleku M., Keil O., Endruschat J., Esche V., Durieux B., Löffler K., Fechtner M., Röhl T., Fisch G. RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy. Gene Ther., 2006 13 1360-1370.
77. Santel A., Aleku M., Keil O., Endruschat J., Esche V., Fisch G., Dames S., Löffler K., Fechtner M., Arnold W. A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium. Gene Ther., 2006 13 1222-1234.
78. Aleku M., Schulz P., Keil O., Santel A., Schaeper U., Dieckhoff B., Janke O., Endruschat J., Durieux B., Röder N. Atu027 a liposomal small interfering RNA formulation targeting protein kinase N3 inhibits cancer progression. Cancer Res., 2008 68 9788-9798.
79. Santel A., Aleku M., Röder N., Möpert K., Durieux B., Janke O., Keil O., Endruschat J., Dames S., Lange C. Atu027 prevents pulmonary metastasis in experimental and spontaneous mouse metastasis models. Clin. Cancer Res., 2010 16 5469-5480.
80. Strumberg D., Schultheis B., Traugott U., Vank C., Santel A., Keil O., Giese K., Kaufmann J., Drevs J. Phase I clinical development of Atu027 a siRNA formulation targeting PKN3 in patients with advanced solid tumors. Int. J. Clin. Pharmacol. Ther., 2012 50 76.
81. Jayaraman M., Ansell S.M., Mui B.L., Tam Y.K., Chen J., Du X., Butler D., Eltepu L., Matsuda S., Narayanannair J.K. Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo. Angew. Chem. Int. Ed., 2012 51 8529-8533.
82. Coelho T., Adams D., Silva A., Lozeron P., Hawkins P.N., Mant T., Perez J., Chiesa J., Warrington S., Tranter E. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. New England J. Med., 2013 369 819-829.